List of works by James Wilson

"Stealth" adenoviruses blunt cell-mediated and humoral immune responses against the virus and allow for significant gene expression upon readministration in the lung

scientific article

2017 Is the Year We Have Been Waiting For.

scientific article published in December 2017

31st Annual Meeting and Associated Programs of the Society for Immunotherapy of Cancer (SITC 2016): part two: National Harbor, MD, USA. 9-13 November 2016

scientific article published oln 16 November 2016

5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency.

scientific article published on 10 April 2017

A Birds-Eye View: An Interview with Nick Leschly

scientific article published on 01 March 2019

A CD46-binding chimpanzee adenovirus vector as a vaccine carrier.

scientific article published on 16 January 2007

A Gene Therapy Approach to Improve Copper Metabolism and Prevent Liver Damage in a Mouse Model of Wilson Disease

scientific article published on 25 February 2019

A Journey in the Development of Gene Therapy for Inherited Disorders of the Bone Marrow.

scientific article

A call to arms for improved vector analytics!

scientific article published on 01 February 2015

A common mechanism for cytoplasmic dynein-dependent microtubule binding shared among adeno-associated virus and adenovirus serotypes

scientific article

A comparative analysis of novel fluorescent proteins as reporters for gene transfer studies.

scientific article published on 17 May 2007

A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice

scientific article published on February 2016

A molecular survey of hypoxanthine-guanine phosphoribosyltransferase deficiency in man.

scientific article

A mutation-independent CRISPR-Cas9-mediated gene targeting approach to treat a murine model of ornithine transcarbamylase deficiency

scientific article published on 12 February 2020

A new editorial team

scientific article published on 01 July 2011

A new genetic vaccine platform based on an adeno-associated virus isolated from a rhesus macaque

scientific article published on 7 October 2009

A new scalable method for the purification of recombinant adenovirus vectors.

scientific article published in November 2002

A pilot study of in vivo liver-directed gene transfer with an adenoviral vector in partial ornithine transcarbamylase deficiency

scientific article

A pilot study of systemic corticosteroid administration in conjunction with intrapleural adenoviral vector administration in patients with malignant pleural mesothelioma.

scientific article published in December 2000

A quantitative nonimmunogenic transgene product for evaluating vectors in nonhuman primates.

scientific article published in December 2000

A randomised, double-blind, placebo-controlled trial of repeated nebulisation of non-viral cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy in patients with cystic fibrosis

scientific article

A replication-defective human adenovirus recombinant serves as a highly efficacious vaccine carrier.

scientific article published on May 1996

A simian replication-defective adenoviral recombinant vaccine to HIV-1 gag.

scientific article published on February 2003

A single injection of an optimized AAV vector into cerebrospinal fluid corrects neurological disease in a murine model of GM1 gangliosidosis

scientific article published on 12 October 2020

A single-step affinity column for purification of serotype-5 based adeno-associated viral vectors.

scientific article

A welcome animal model

scientific article published in Nature

AAV Natural Infection Induces Broad Cross-Neutralizing Antibody Responses to Multiple AAV Serotypes in Chimpanzees

scientific article published on June 2016

AAV as an immunogen

scientific article published on October 2007

AAV gene therapy corrects OTC deficiency and prevents liver fibrosis in aged OTC-knock out heterozygous mice.

scientific article published on 2 March 2017

AAV vectors avoid inflammatory signals necessary to render transduced hepatocyte targets for destructive T cells

scientific article

AAV vectors expressing LDLR gain-of-function variants demonstrate increased efficacy in mouse models of familial hypercholesterolemia

scientific article published on 14 July 2014

AAV2 vector harboring a liver-restricted promoter facilitates sustained expression of therapeutic levels of alpha-galactosidase A and the induction of immune tolerance in Fabry mice.

scientific article published in February 2004

AAV8 Gene Therapy Rescues the Newborn Phenotype of a Mouse Model of Crigler-Najjar

scientific article published on 16 February 2018

AAV8 Gene Therapy for Crigler-Najjar Syndrome in Macaques Elicited Transgene T Cell Responses That Are Resident to the Liver

article

AAV8 capsid variable regions at the two-fold symmetry axis contribute to high liver transduction by mediating nuclear entry and capsid uncoating.

scientific article published on 13 March 2014

AAV8 induces tolerance in murine muscle as a result of poor APC transduction, T cell exhaustion, and minimal MHCI upregulation on target cells.

scientific article published on 19 June 2013

AAV8-antiVEGFfab Ocular Gene Transfer for Neovascular Age-Related Macular Degeneration.

scientific article published on 8 December 2017

AAV8-mediated hepatic gene transfer in infant rhesus monkeys (Macaca mulatta)

scientific article published on 2 August 2011

AAV9 targets cone photoreceptors in the nonhuman primate retina

scientific article

Ablation of E2A in recombinant adenoviruses improves transgene persistence and decreases inflammatory response in mouse liver

scientific article published on June 1994

Abnormal polyamine metabolism is unique to the neuropathic forms of MPS: potential for biomarker development and insight into pathogenesis

scientific article published in October 2017

Absolute determination of single-stranded and self-complementary adeno-associated viral vector genome titers by droplet digital PCR.

scientific article published on 14 February 2014

Accurate and Rapid Sequence Analysis of Adeno-Associated Virus Plasmids by Illumina Next-Generation Sequencing

scientific article published on 22 August 2018

Activation of CFTR-specific T Cells in cystic fibrosis mice following gene transfer.

scientific article published on 19 June 2007

Activation of innate immunity in nonhuman primates following intraportal administration of adenoviral vectors.

scientific article

Activation of transgene-specific T cells following lentivirus-mediated gene delivery to mouse lung

scientific article published on September 2009

Adeno-Associated Virus Serotype 9-Expressed ZMapp in Mice Confers Protection Against Systemic and Airway-Acquired Ebola Virus Infection

scientific article

Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology

scientific article published on 31 July 2020

Adeno-associated viral gene therapy corrects a mouse model of argininosuccinic aciduria

article

Adeno-associated virus (AAV) serotype 9 provides global cardiac gene transfer superior to AAV1, AAV6, AAV7, and AAV8 in the mouse and rat.

scientific article

Adeno-associated virus 9-mediated airway expression of antibody protects old and immunodeficient mice against influenza virus

scientific article

Adeno-associated virus and lentivirus pseudotypes for lung-directed gene therapy

scientific article

Adeno-associated virus antibody profiles in newborns, children, and adolescents

scientific article

Adeno-associated virus capsid structure drives CD4-dependent CD8+ T cell response to vector encoded proteins

scientific article published in May 2009

Adeno-associated virus mediates long-term gene transfer and delivery of chondroprotective IL-4 to murine synovium.

scientific article

Adeno-associated virus serotype 1-based gene therapy for FTD caused by GRN mutations

scientific article published on 16 September 2020

Adeno-associated virus serotype 8 gene therapy leads to significant lowering of plasma cholesterol levels in humanized mouse models of homozygous and heterozygous familial hypercholesterolemia

scientific article published on 14 November 2012

Adeno-associated virus serotype 9 vectors transduce murine alveolar and nasal epithelia and can be readministered.

scholarly article

Adeno-associated virus-mediated expression of human butyrylcholinesterase to treat organophosphate poisoning

scientific article published on 25 November 2019

Adeno-associated virus-mediated gene transfer to nonhuman primate liver can elicit destructive transgene-specific T cell responses.

scientific article

Adeno-associated viruses undergo substantial evolution in primates during natural infections

scientific article

Adenosine deaminase deficiency with normal immune function. An acidic enzyme mutation.

scientific article

Adenoviral vector-mediated gene therapy in the mouse lung: no role of Fas-Fas ligand interactions for elimination of transgene expression in bronchioepithelial cells.

scientific article published in November 1999

Adenoviral-mediated gene transfer to rabbit synovium in vivo.

scientific article

Adenovirus-Antibody Complexes Contributed to Lethal Systemic Inflammation in a Gene Therapy Trial

scientific article published on 07 February 2020

Adenovirus-adeno-associated virus hybrid for large-scale recombinant adeno-associated virus production

scientific article published on September 2009

Adenovirus-based genetic vaccines for biodefense

scientific article

Adenovirus-based vaccine prevents pneumonia in ferrets challenged with the SARS coronavirus and stimulates robust immune responses in macaques

article

Adenovirus-based vaccines generate cytotoxic T lymphocytes to epitopes of NS1 from dengue virus that are present in all major serotypes

scientific article

Adenovirus-mediated gene transfer to liver

scientific article

Adenovirus-mediated herpes simplex virus thymidine kinase/ganciclovir gene therapy in patients with localized malignancy: results of a phase I clinical trial in malignant mesothelioma.

scientific article published in May 1998

Adenovirus-mediated transfer of the CFTR gene to lung of nonhuman primates: biological efficacy study.

scientific article published in December 1993

Adenoviruses in fecal samples from asymptomatic rhesus macaques, United States ⬇️

scientific article

Adenoviruses in lymphocytes of the human gastro-intestinal tract

scientific article (publication date: 2011)

Advancing translational research through the NHLBI Gene Therapy Resource Program (GTRP).

scientific article published on March 2013

Adverse events in gene transfer trials and an agenda for the new year

scientific article published in January 2008

Airway epithelia regulate expression of human beta-defensin 2 through Toll-like receptor 2.

scientific article published on 3 July 2003

Alternative Start Sites Downstream of Non-Sense Mutations Drive Antigen Presentation and Tolerance Induction to C-Terminal Epitopes

scientific article published on 12 May 2017

Amelioration of collagen-induced arthritis by CD95 (Apo-1/Fas)-ligand gene transfer.

scientific article published on October 1997

An AAV vector-mediated gene delivery approach facilitates reconstitution of functional human CD8+ T cells in mice

scientific article

An optimized protocol for detection of E. coli beta-galactosidase in lung tissue following gene transfer.

scientific article

Analysis of particle content of recombinant adeno-associated virus serotype 8 vectors by ion-exchange chromatography.

scientific article published on February 2012

Analysis of tumors arising in male B6C3F1 mice with and without AAV vector delivery to liver.

scientific article published on 6 May 2006

Animal models of human disease for gene therapy

scientific article published on March 1996

Assessment of Humoral, Innate, and T-Cell Immune Responses to Adeno-Associated Virus Vectors

article

Assessment of humoral, innate, and T-cell immune responses to adeno-associated virus vectors.

scientific article

Autoimmunity, recessive diseases, and gene replacement therapy.

scientific article published on December 2010

BALB/c mice show impaired hepatic tolerogenic response following AAV gene transfer to the liver

scientific article

Bacterial phosphorylcholine decreases susceptibility to the antimicrobial peptide LL-37/hCAP18 expressed in the upper respiratory tract.

scientific article published on March 2000

Biodistribution of AAV8 vectors expressing human low-density lipoprotein receptor in a mouse model of homozygous familial hypercholesterolemia

scientific article

Biology of AAV serotype vectors in liver-directed gene transfer to nonhuman primates.

scientific article

Biology of E1-deleted adenovirus vectors in nonhuman primate muscle.

scientific article published on June 2001

Blunting of immune responses to adenoviral vectors in mouse liver and lung with CTLA4Ig.

scientific article

Breakthrough to Bedside: Bringing Gene Therapy to Neuromuscular Diseases

scientific article published on 10 September 2019

Bulls, bubbles, and biotech

scientific article published in August 2013

CD40 ligand-dependent T cell activation: requirement of B7-CD28 signaling through CD40.

scientific article

CRISPR/Cas9 directed to the Ube3a antisense transcript improves Angelman syndrome phenotype in mice

scientific article published on 07 January 2021

CRISPR/Cas9-mediated in vivo gene targeting corrects hemostasis in newborn and adult factor IX-knockout mice

scientific article published on 11 April 2019

Cardiac gene transfer of short hairpin RNA directed against phospholamban effectively knocks down gene expression but causes cellular toxicity in canines

scientific article

Carl June Speaks of His Pioneering Efforts That Led to the First Food and Drug Administration-Approved Gene Therapy Product

scientific article published on 13 November 2017

Cathelicidins--a family of multifunctional antimicrobial peptides.

scientific article

Cationic lipid formulations alter the in vivo tropism of AAV2/9 vector in lung

scientific article

Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy

scientific article

Challenges in the gene therapy commercial ecosystem

scientific article published in September 2017

Characterization of Adeno-Associated Viral Vector-Mediated Human Factor VIII Gene Therapy in Hemophilia A Mice

scientific article published on 5 January 2017

Characterization of a family of chimpanzee adenoviruses and development of molecular clones for gene transfer vectors.

scientific article published on May 2004

Chimpanzee adenovirus CV-68 adapted as a gene delivery vector interacts with the coxsackievirus and adenovirus receptor

scientific article published in January 2002

Chimpanzee adenovirus vaccine protects against Zaire Ebola virus

scientific article

Chimpanzee-origin adenovirus vectors as vaccine carriers.

scientific article published on March 2006

Chronic suppression of heart-failure progression by a pseudophosphorylated mutant of phospholamban via in vivo cardiac rAAV gene delivery

scientific article

Clades of Adeno-associated viruses are widely disseminated in human tissues.

scientific article

Class I-restricted T-cell responses to a polymorphic peptide in a gene therapy clinical trial for α-1-antitrypsin deficiency.

scientific article published on 30 January 2017

Combination Adenovirus and Protein Vaccines Prevent Infection or Reduce Viral Burden after Heterologous Clade C Simian-Human Immunodeficiency Virus Mucosal Challenge

scientific article published in November 2017

Combination therapy with lamivudine and adenovirus causes transient suppression of chronic woodchuck hepatitis virus infections.

scientific article

Combined 5-fluorouracil/systemic interferon-beta gene therapy results in long-term survival in mice with established colorectal liver metastases

scientific article published on February 2004

Comparative Study of Liver Gene Transfer With AAV Vectors Based on Natural and Engineered AAV Capsids.

scientific article

Comparative analysis of adeno-associated viral vector serotypes 1, 2, 5, 7, and 8 in mouse brain

scientific article published in March 2007

Complete deficiency of the low-density lipoprotein receptor is associated with increased apolipoprotein B-100 production.

scientific article published on 6 January 2005

Complete nucleotide sequences and genome organization of four chimpanzee adenoviruses.

scientific article published on July 2004

Complete prevention of atherosclerosis in apoE-deficient mice by hepatic human apoE gene transfer with adeno-associated virus serotypes 7 and 8.

scientific article published on 8 June 2006

Construction of gene transfer vectors based on simian adenovirus 7.

scientific article published on 11 May 2011

Context-Specific Function of the Engineered Peptide Domain of PHP.B.

scientific article published on 04 August 2021

Correction of the cystic fibrosis defect in vitro by retrovirus-mediated gene transfer ⬇️

scientific article published on September 21, 1990

Correction of the dystrophic phenotype by in vivo targeting of muscle progenitor cells

scientific article published in October 2003

Correction of the genetic defect in hepatocytes from the Watanabe heritable hyperlipidemic rabbit.

scientific article

Correction of ureagenesis after gene transfer in an animal model and after liver transplantation in humans with ornithine transcarbamylase deficiency.

scientific article published in November 1999

Correction to: ITR-Seq, a next-generation sequencing assay, identifies genome-wide DNA editing sites in vivo following adeno-associated viral vector-mediated genome editing

scientific article published on 20 November 2020

Correction: AAV9 Targets Cone Photoreceptors in the Nonhuman Primate Retina.

scientific article published on 8 August 2013

Corrigendum to "Biology of AAV Serotype Vectors in Liver-Directed Gene Transfer to Nonhuman Primates".

scientific article published on 30 May 2006

CpG-depleted adeno-associated virus vectors evade immune detection.

scientific article published on 17 June 2013

Creatine kinase, a magnetic resonance-detectable marker gene for quantification of liver-directed gene transfer.

scientific article published in December 2005

Creation of a panel of vectors based on ape adenovirus isolates

scientific article published on 17 December 2010

Cross-presentation of adeno-associated virus serotype 2 capsids activates cytotoxic T cells but does not render hepatocytes effective cytolytic targets

scientific article published on March 2007

Cycling at the Frontiers of Gene Therapy

scientific article published on 01 June 2019

Cyclophosphamide diminishes inflammation and prolongs transgene expression following delivery of adenoviral vectors to mouse liver and lung.

scientific article

Cystic fibrosis. More from the modellers

scientific article published on September 17, 1992

Cystic fibrosis. Vehicles for gene therapy.

scientific article published in October 1993

Deamidation of Amino Acids on the Surface of Adeno-Associated Virus Capsids Leads to Charge Heterogeneity and Altered Vector Function

scientific article published on 18 October 2018

Delivery of adeno-associated virus vectors to the fetal retina: impact of viral capsid proteins on retinal neuronal progenitor transduction

scientific article

Delivery of an Adeno-Associated Virus Vector into Cerebrospinal Fluid Attenuates Central Nervous System Disease in Mucopolysaccharidosis Type II Mice.

scientific article published on 10 August 2016

Detection Assay for Replication-Competent Adenovirus by Concentration Passage and Real-Time Quantitative Polymerase Chain Reaction (qPCR)

scientific article published on 02 December 2019

Detection of reporter gene expression in murine airways

scientific article published in January 2007

Determining the Minimally Effective Dose of a Clinical Candidate AAV Vector in a Mouse Model of Crigler-Najjar Syndrome

scientific article published on 21 July 2018

Development and rescue of human familial hypercholesterolaemia in a xenograft mouse model

scientific article

Development of formulations that enhance physical stability of viral vectors for gene therapy.

scientific article published in September 2001

Development of novel formulations that enhance adenoviral-mediated gene expression in the lung in vitro and in vivo.

scientific article published in July 2001

Diet-dependent cardiovascular lipid metabolism controlled by hepatic LXRalpha

scientific article published in May 2005

Direct gene transfer of human CFTR into human bronchial epithelia of xenografts with E1-deleted adenoviruses.

scientific article published on May 1993

Dosage thresholds for AAV2 and AAV8 photoreceptor gene therapy in monkey

scientific article

Dual reporter comparative indexing of rAAV pseudotyped vectors in chimpanzee airway.

scientific article published on 13 October 2009

Effect of blood collection technique in mice on clinical pathology parameters

scientific article published in January 2002

Effect of preexisting immunity on an adenovirus vaccine vector: in vitro neutralization assays fail to predict inhibition by antiviral antibody in vivo.

scientific article published on 11 March 2009

Effects of Self-Complementarity, Codon Optimization, Transgene, and Dose on Liver Transduction with AAV8.

scientific article

Efficacy of severe acute respiratory syndrome vaccine based on a nonhuman primate adenovirus in the presence of immunity against human adenovirus

scientific article published in May 2006

Efficient gene transfer into the mouse lung by fetal intratracheal injection of rAAV2/6.2.

scientific article published on 27 July 2010

Efficient serotype-dependent release of functional vector into the culture medium during adeno-associated virus manufacturing.

scientific article published on October 2010

Efficient trans-splicing in the retina expands the utility of adeno-associated virus as a vector for gene therapy.

scientific article

Efficient transduction of human monocyte-derived dendritic cells by chimpanzee-derived adenoviral vector.

scientific article published in April 2003

Efficient transduction of liver and muscle after in utero injection of lentiviral vectors with different pseudotypes

scientific article published on September 2002

Enhanced survival of the LINCL mouse following CLN2 gene transfer using the rh.10 rhesus macaque-derived adeno-associated virus vector.

scientific article published on 19 December 2006

Enhancing the utility of adeno-associated virus gene transfer through inducible tissue-specific expression

scientific article published on August 2013

Erythropoietin gene therapy leads to autoimmune anemia in macaques

scientific article published on 24 December 2003

Evaluating the potential of germ line transmission after intravenous administration of recombinant adenovirus in the C3H mouse.

scientific article published in September 1998

Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII.

scientific article

Evaluation of adeno-associated viral vectors for liver-directed gene transfer in dogs.

scientific article

Evaluation of an E1E4-deleted adenovirus expressing the herpes simplex thymidine kinase suicide gene in cancer gene therapy.

scientific article published in February 1999

Evaluation of intrathecal routes of administration for adeno-associated virus vectors in large animals.

scientific article published on 15 August 2017

Evaluation of toxicity from high-dose systemic administration of recombinant adenovirus vector in vector-naive and pre-immunized mice.

scientific article

Ex vivo gene therapy of familial hypercholesterolemia.

scientific article published in April 1992

Expanded repertoire of AAV vector serotypes mediate unique patterns of transduction in mouse brain.

scientific article

Expression of Retroviral Transduced Human CD18 in Murine Cells: AnIn VitroModel of Gene Therapy for Leukocyte Adhesion Deficiency ⬇️

scientific article published on January 1, 1991

Expression of an abundant alternatively spliced form of the cystic fibrosis transmembrane conductance regulator (CFTR) gene is not associated with a cAMP-activated chloride conductance.

scientific article published in March 1993

Expression of the cystic fibrosis gene in adult human lung

scientific article

Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer

scientific article

Flushing out antibodies to make AAV gene therapy available to more patients

scientific article published on February 2013

Formation of newly synthesized adeno-associated virus capsids in the cell nucleus

scientific article published on June 2014

Functional cystic fibrosis transmembrane conductance regulator expression in cystic fibrosis airway epithelial cells by AAV6.2-mediated segmental trans-splicing

scientific article

Gendicine: the first commercial gene therapy product

scientific article

Gene Therapy Entering the Land of Oz

scientific article published on 01 December 2018

Gene Therapy in a Humanized Mouse Model of Familial Hypercholesterolemia Leads to Marked Regression of Atherosclerosis ⬇️

scientific article (publication date: 19 October 2010)

Gene therapy for cystic fibrosis using E1-deleted adenovirus: a phase I trial in the nasal cavity. The University of North Carolina at Chapel Hill.

scientific article published on May 1994

Gene therapy for dyslipidemia: a review of gene replacement and gene inhibition strategies

scientific article published in June 2010

Gene therapy for mucopolysaccharidosis type VI is effective in cats without pre-existing immunity to AAV8.

scientific article published on 30 January 2013

Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012

scientific article (publication date: April 2013)

Gene therapy in a xenograft model of cystic fibrosis lung corrects chloride transport more effectively than the sodium defect.

scientific article published in February 1995

Gene therapy of hypercholesterolemic disorders.

scientific article published in September 1995

Gene therapy with novel adeno-associated virus vectors substantially diminishes atherosclerosis in a murine model of familial hypercholesterolemia

scientific article

Gene therapy: adenovirus vectors.

scientific article

Gene therapy: socioeconomic and ethical issues. A roundtable discussion.

scientific article published in June 1996

Gene transfer in human skin with different pseudotyped HIV-based vectors.

scientific article published on February 2007

Gene transfer into skeletal muscle using novel AAV serotypes.

scientific article

Gene transfer of wild-type apoA-I and apoA-I Milano reduce atherosclerosis to a similar extent

scientific article published on 2 May 2007

Generation of a mouse expressing a conditional knockout of the hepatocyte growth factor gene: demonstration of impaired liver regeneration

scientific article published in September 2004

Generation of an adenoviral vaccine vector based on simian adenovirus 21.

scientific article

Genetic diseases, immunology, viruses, and gene therapy

scientific article

Good news on the clinical gene transfer front

scientific article published on 01 May 2008

HCO3(-) secretion by murine nasal submucosal gland serous acinar cells during Ca2+-stimulated fluid secretion.

scientific article published on July 2008

HIV-1 suppression and durable control by combining single broadly neutralizing antibodies and antiretroviral drugs in humanized mice

scientific article published on 16 September 2013

Helper lipid structure influences protein adsorption and delivery of lipid nanoparticles to spleen and liver

scientific article published on 06 January 2021

Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid

scientific article published on 16 July 2006

Hepatic gene transfer in neonatal mice by adeno-associated virus serotype 8 vector.

scientific article

Hepatic regulatory T cells and Kupffer cells are crucial mediators of systemic T cell tolerance to antigens targeting murine liver.

scientific article published on August 2009

Hepatocellular carcinoma in a research subject with ornithine transcarbamylase deficiency

scientific article published on 7 November 2011

Hepatocyte transplantation: development of new systems for liver repopulation and gene therapy

scientific article published on August 1, 1992

High levels of persistent expression of alpha1-antitrypsin mediated by the nonhuman primate serotype rh.10 adeno-associated virus despite preexisting immunity to common human adeno-associated viruses

scientific article

High throughput creation of recombinant adenovirus vectors by direct cloning, green-white selection and I-Sce I-mediated rescue of circular adenovirus plasmids in 293 cells ⬇️

scientific article published on October 1, 2003

High throughput method for creating and screening recombinant adenoviruses.

scientific article published in August 1998

High-level transgene expression in nonhuman primate liver with novel adeno-associated virus serotypes containing self-complementary genomes

scientific article published on June 2006

Host immune responses to chronic adenovirus infections in human and nonhuman primates.

scientific article published on 30 December 2008

Human CRB1-associated retinal degeneration: comparison with the rd8 Crb1-mutant mouse model

scientific article

Human Gene Therapy Clinical Development: Where the Academy and Industry Meet

scientific article

Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression

scientific article

Human adenine phosphoribosyltransferase: characterization from subjects with a deficiency of enzyme activity

scientific article published on 01 December 1983

Human airway epithelial cells sense Pseudomonas aeruginosa infection via recognition of flagellin by Toll-like receptor 5.

scientific article

Human beta-defensin-1 is a salt-sensitive antibiotic in lung that is inactivated in cystic fibrosis

scientific article

Human cone photoreceptor dependence on RPE65 isomerase

scientific article

Human cystic fibrosis transmembrane conductance regulator directed to respiratory epithelial cells of transgenic mice

scientific article published on 01 September 1992

Human fetal trachea-SCID mouse xenografts: efficacy of vesicular stomatitis virus-G pseudotyped lentiviral-mediated gene transfer.

scientific article published on June 2003

Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics

scholarly article

Human hypoxanthine-guanine phosphoribosyltransferase. Demonstration of structural variants in lymphoblastoid cells derived from patients with a deficiency of the enzyme

scientific article published on March 1982

Human hypoxanthine-guanine phosphoribosyltransferase. Detection of a mutant allele by restriction endonuclease analysis.

scientific article

Human immune system mice immunized with Plasmodium falciparum circumsporozoite protein induce protective human humoral immunity against malaria

scientific article published on 24 September 2015

Human immunodeficiency viral vector pseudotyped with the spike envelope of severe acute respiratory syndrome coronavirus transduces human airway epithelial cells and dendritic cells.

scientific article published on May 2007

Human immunodeficiency virus type 1-specific immune responses in primates upon sequential immunization with adenoviral vaccine carriers of human and simian serotypes

scientific article

Humility and clinical trials

scientific article published on 01 September 2007

Humoral Immune Response to AAV.

scientific article published on 18 October 2013

Humoral and cell-mediated immune response, and growth factor synthesis after direct intraarticular injection of rAAV2-IGF-I and rAAV5-IGF-I in the equine middle carpal joint.

scientific article published on March 2015

Hypoxanthine-guanine phosphoribosyltransferase. Genetic evidence for identical mutations in two partially deficient subjects

scientific article published on December 1988

ITR-Seq, a next-generation sequencing assay, identifies genome-wide DNA editing sites in vivo following adeno-associated viral vector-mediated genome editing

scientific article published on 17 March 2020

Identification and functional characterization in vivo of a novel splice variant of LDLR in rhesus macaques

scientific article (publication date: 16 August 2011)

Identification of an adeno-associated virus binding epitope for AVB sepharose affinity resin

scientific article published on 4 November 2015

Identification of murine CD8 T cell epitopes in codon-optimized SARS-associated coronavirus spike protein

scientific article

Identification of the galactose binding domain of the adeno-associated virus serotype 9 capsid

scientific article

Identification of the immunodominant cytotoxic T-cell epitope of human alpha-1 antitrypsin.

scientific article

Identification of the murine AAVrh32.33 capsid-specific CD8+ T cell epitopes

scientific article published in December 2009

Identification of the murine firefly luciferase-specific CD8 T-cell epitopes.

scientific article

Immune responses to adenovirus and adeno-associated virus in humans.

scientific article

Immunogenicity in Mamu-A*01 rhesus macaques of a CCR5-tropic human immunodeficiency virus type 1 envelope from the primary isolate (Bx08) after synthetic DNA prime and recombinant adenovirus 5 boost

scientific article published on January 2003

Immunology of gene therapy with adenoviral vectors in mouse skeletal muscle.

scientific article

Impact of intravenous infusion time on AAV8 vector pharmacokinetics, safety, and liver transduction in cynomolgus macaques

scientific article published on 7 December 2016

Impact of pre-existing immunity on gene transfer to nonhuman primate liver with adeno-associated virus 8 vectors

scientific article published on 8 June 2011

Impact of preexisting and induced humoral and cellular immune responses in an adenovirus-based gene therapy phase I clinical trial for localized mesothelioma.

scientific article published in September 1998

Impact of preexisting vector immunity on the efficacy of adeno-associated virus-based HIV-1 Gag vaccines.

scientific article published on July 2008

Implantation of vascular grafts lined with genetically modified endothelial cells

scientific article published on 01 June 1989

In memory of Sonia Skarlatos, PhD (1953-2013)

scientific article

In the Beginning: Reflections on the Genesis of Molecular Therapy

scientific article published on January 2010

In vivo detection of gene expression in liver by 31P nuclear magnetic resonance spectroscopy employing creatine kinase as a marker gene

scientific article

In vivo evaluation of adeno-associated virus gene transfer in airways of mice with acute or chronic respiratory infection

scientific article

In vivo measurement of ureagenesis with stable isotopes

scientific article published on 01 January 1998

In vivo quantitative noninvasive imaging of gene transfer by single-photon emission computerized tomography.

scientific article published in February 2003

In vivo retroviral gene transfer into human bronchial epithelia of xenografts

scientific article published on December 1992

In vivo selection of hepatocytes transduced with adeno-associated viral vectors.

scientific article published in May 2000

In vivo somatic cell gene transfer of an engineered Noggin mutein prevents BMP4-induced heterotopic ossification

scientific article published in December 2003

Inactivation of E2a in recombinant adenoviruses improves the prospect for gene therapy in cystic fibrosis.

scientific article published in July 1994

Increased mucosal CD4+ T cell activation in rhesus macaques following vaccination with an adenoviral vector

scientific article published on 14 May 2014

Increased mucosal CD4+ T-cell activation following vaccination with an adenoviral vector in rhesus macaques

Increasing the specificity of AAV-based gene editing through self-targeting and short promoter strategies

scientific article published on 24 December 2020

Induction of CD8+ T cells to an HIV-1 antigen through a prime boost regimen with heterologous E1-deleted adenoviral vaccine carriers.

scientific article published on December 2003

Induction of CD8+ T cells to an HIV-1 antigen upon oral immunization of mice with a simian E1-deleted adenoviral vector

scientific article published on 01 January 2004

Induction of protective immunity to anthrax lethal toxin with a nonhuman primate adenovirus-based vaccine in the presence of preexisting anti-human adenovirus immunity.

scientific article

Inefficient gene transfer by adenovirus vector to cystic fibrosis airway epithelia of mice and humans ⬇️

scientific article published on October 27, 1994

Inflammation promotes the loss of adeno-associated virus-mediated transgene expression in mouse liver.

scientific article

Influence of serotype, cell type, tissue composition, and time after inoculation on gene expression in recombinant adeno-associated viral vector-transduced equine joint tissues

scientific article

Inhibition of retinal neovascularization by intraocular viral-mediated delivery of anti-angiogenic agents

scientific article published on 01 October 2002

Innate immune responses to adenoviral vector-mediated acute pancreatitis.

scientific article published in March 2005

Interview with Inder Verma, PhD.

scientific article published in March 2016

Interview with Jean Bennett, MD, PhD

scientific article published on 01 March 2018

Interview with Katherine A. High, MD.

scientific article published in December 2016

Intra-arterial delivery of a recombinant adenovirus does not increase gene transfer to tumor cells in a rat model of metastatic colorectal carcinoma

article

Intracranial administration of adenovirus expressing HSV-TK in combination with ganciclovir produces a dose-dependent, self-limiting inflammatory response.

scientific article

Intramuscular administration of AAV overcomes pre-existing neutralizing antibodies in rhesus macaques.

scientific article

Intramuscular injection of AAV8 in mice and macaques is associated with substantial hepatic targeting and transgene expression

scientific article

Intranasal antibody gene transfer in mice and ferrets elicits broad protection against pandemic influenza

scientific article

Intrathecal Viral Vector Delivery of Trastuzumab Prevents or Inhibits Tumor Growth of Human HER2-Positive Xenografts in Mice

scientific article published on 28 August 2018

Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis I

scientific article published on 16 July 2014

Inverse zonation of hepatocyte transduction with AAV vectors between mice and non-human primates.

scientific article published on 12 June 2011

Ionizable lipid nanoparticles encapsulating barcoded mRNA for accelerated in vivo delivery screening

scientific article published on 31 October 2019

Isolating Human Monoclonal Antibodies Against Adeno-Associated Virus From Donors With Pre-existing Immunity

scientific article published on 07 July 2020

Isolation and characterization of adenoviruses persistently shed from the gastrointestinal tract of non-human primates

scientific article published on 3 July 2009

Isolation of highly infectious and pure adeno-associated virus type 2 vectors with a single-step gravity-flow column.

scientific article

It's time for gene therapy to get disruptive!

scientific article published in January 2012

Jurassic Park, Gene Therapy, and Neuroscience: An Interview with Feng Zhang, PhD.

scientific article published on 6 March 2017

Lentiviral vectors for gene therapy of cystic fibrosis.

scientific article

Lentiviral vectors pseudotyped with minimal filovirus envelopes increased gene transfer in murine lung ⬇️

scientific article published on November 1, 2003

Lessons learned from the clinical development and market authorization of Glybera.

scientific article published on 29 June 2013

Lessons learned from the gene therapy trial for ornithine transcarbamylase deficiency

scientific article

Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type I

scientific article

Long-term correction of ammonia metabolism and prolonged survival in ornithine transcarbamylase-deficient mice following liver-directed treatment with adeno-associated viral vectors.

scientific article published on 3 May 2006

Long-term follow-up of patients with malignant pleural mesothelioma receiving high-dose adenovirus herpes simplex thymidine kinase/ganciclovir suicide gene therapy

scientific article published on October 2005

Long-term inducible gene expression in the eye via adeno-associated virus gene transfer in nonhuman primates.

scientific article published in February 2005

Long-term pharmacologically regulated expression of erythropoietin in primates following AAV-mediated gene transfer.

scientific article

Long-term regulated expression of growth hormone in mice after intramuscular gene transfer

scientific article published on July 1999

Long-term restoration of cardiac dystrophin expression in golden retriever muscular dystrophy following rAAV6-mediated exon skipping

scientific article published on 6 December 2011

Longitudinal evaluation and assessment of cardiovascular disease in patients with homozygous familial hypercholesterolemia

scientific article

Luigi Naldini on His Lifelong Involvement with the Development of Gene Therapy

scientific article published on 24 August 2016

Lung homing CTLs and their proliferation ability are important correlates of vaccine protection against influenza

scientific article published on 29 June 2010

MHC class I-restricted cytotoxic T lymphocytes to viral antigens destroy hepatocytes in mice infected with E1-deleted recombinant adenoviruses.

scientific article published on August 1994

MR and fluorescent imaging of low-density lipoprotein receptors.

scientific article

Macaque model for severe acute respiratory syndrome

scientific article published on October 2004

Mapping an Adeno-associated Virus 9-Specific Neutralizing Epitope To Develop Next-Generation Gene Delivery Vectors

article

Mapping the structural determinants responsible for enhanced T cell activation to the immunogenic adeno-associated virus capsid from isolate rhesus 32.33.

scientific article published on 29 May 2013

Measuring the Infectious Titer of Recombinant Adenovirus Using Tissue Culture Infection Dose 50% (TCID50) End-Point Dilution and Quantitative Polymerase Chain Reaction (qPCR)

scientific article published on 01 August 2019

Medicine. A history lesson for stem cells

scientific article published on 01 May 2009

Meganuclease targeting of PCSK9 in macaque liver leads to stable reduction in serum cholesterol

scientific article published on 9 July 2018

Methods of gene delivery.

scientific article

MicroRNA-mediated inhibition of transgene expression reduces dorsal root ganglion toxicity by AAV vectors in primates

scientific article published on 01 November 2020

Molecular analysis of vector genome structures after liver transduction by conventional and self-complementary adeno-associated viral serotype vectors in murine and nonhuman primate models

scientific article

Molecular basis of defective anion transport in L cells expressing recombinant forms of CFTR ⬇️

scientific article published on August 1, 1993

Motor neuron transduction after intracisternal delivery of AAV9 in a cynomolgus macaque.

scientific article published on April 2015

Moving forward after two deaths in a gene therapy trial of myotubular myopathy

scientific article published on 30 June 2020

Moving to the clinic with gene therapy through our new journal expansion, human gene therapy clinical development

scientific article

Mucosal delivery of adenovirus-based vaccine protects against Ebola virus infection in mice

scientific article

Mucosally Delivered E1-Deleted Adenoviral Vaccine Carriers Induce Transgene Product-Specific Antibody Responses in Neonatal Mice ⬇️

scientific article published on October 15, 2003

Multiple recombinant adeno-associated viral vector serotypes display persistent in vivo gene expression in vector-transduced rat stifle joints

scientific article published on June 2013

Muscle-directed gene therapy for hemophilia B with more efficient and less immunogenic AAV vectors.

scientific article

Muscle-specific promoters may be necessary for adeno-associated virus-mediated gene transfer in the treatment of muscular dystrophies.

scientific article

Natural killer T cell ligand alpha-galactosylceramide enhances protective immunity induced by malaria vaccines

scientific article

Naturally occurring singleton residues in AAV capsid impact vector performance and illustrate structural constraints.

scientific article published on December 2009

Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates.

scientific article published on 29 May 2015

Neonatal tolerance induction enables accurate evaluation of gene therapy for MPS I in a canine model

scientific article published on 8 June 2016

Neutralizing Antibodies Against Adeno-Associated Viral Capsids in Patients with mut Methylmalonic Acidemia

scientific article published on 20 January 2016

New Candidate Vaccines against Blood-Stage Plasmodium falciparum Malaria: Prime-Boost Immunization Regimens Incorporating Human and Simian Adenoviral Vectors and Poxviral Vectors Expressing an Optimized Antigen Based on Merozoite Surface Protein 1

scholarly article by Anna L. Goodman et al published 19 April 2011 in Infection and Immunity

New candidate vaccines against blood-stage Plasmodium falciparum malaria: prime-boost immunization regimens incorporating human and simian adenoviral vectors and poxviral vectors expressing an optimized antigen based on merozoite surface protein 1

scientific article

New recombinant serotypes of AAV vectors.

scientific article

No evidence for tumorigenesis of AAV vectors in a large-scale study in mice.

scientific article

Non-Clinical Study Examining AAV8.TBG.hLDLR Vector-Associated Toxicity in Chow-Fed Wild-Type and LDLR+/- Rhesus Macaques.

scientific article published on March 2017

Nonclinical Pharmacology/Toxicology Study of AAV8.TBG.mLDLR and AAV8.TBG.hLDLR in a Mouse Model of Homozygous Familial Hypercholesterolemia

scientific article published on March 2017

Nonhuman Primate Models for Diabetic Ocular Neovascularization Using AAV2-Mediated Overexpression of Vascular Endothelial Growth Factor

article by Corinna Lebherz et al published April 2005 in Diabetes

Noninvasive gene transfer to the lung for systemic delivery of therapeutic proteins.

scientific article

Novel AAV serotypes for improved ocular gene transfer

scientific article

Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors

scientific article

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

scientific article

Novel adenoviral vectors coding for GFP-tagged wtCFTR and deltaF508-CFTR: characterization of expression and electrophysiological properties in A549 cells.

scientific article published on December 2004

Novel, chimpanzee serotype 68-based adenoviral vaccine carrier for induction of antibodies to a transgene product

scientific article

Optical imaging of Ca2+-evoked fluid secretion by murine nasal submucosal gland serous acinar cells

scientific article published on 24 May 2007

Optimized Adeno-Associated Viral-Mediated Human Factor VIII Gene Therapy in Cynomolgus Macaques

article

Oral Vaccination of Mice with Adenoviral Vectors Is Not Impaired by Preexisting Immunity to the Vaccine Carrier ⬇️

scientific article published on October 1, 2003

PEGylation of E1-deleted adenovirus vectors allows significant gene expression on readministration to liver

scientific article published in October 2002

PEGylation of a vesicular stomatitis virus G pseudotyped lentivirus vector prevents inactivation in serum

scientific article published on January 2004

Partial protection against H5N1 influenza in mice with a single dose of a chimpanzee adenovirus vector expressing nucleoprotein

scientific article

Percutaneous Transendocardial Delivery of Self-complementary Adeno-associated Virus 6 Achieves Global Cardiac Gene Transfer in Canines

article

Percutaneous transendocardial delivery of self-complementary adeno-associated virus 6 achieves global cardiac gene transfer in canines.

scientific article published on 23 September 2008

Perspectives on best practices for gene therapy programs

scientific article

Pharmacological regulation of protein expression from adeno-associated viral vectors in the eye.

scientific article published in August 2002

Pharmacologically regulated regeneration of functional human pancreatic islets.

scientific article published in January 2005

Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim results

scientific article

Physiological modulation of CFTR activity by AMP-activated protein kinase in polarized T84 cells

scientific article

Preclinical evaluation of a clinical candidate AAV8 vector for ornithine transcarbamylase (OTC) deficiency reveals functional enzyme from each persisting vector genome

scientific article published on 7 November 2011

Prediction of cellular immune responses against CFTR in patients with cystic fibrosis after gene therapy.

scientific article published on 11 January 2007

Preexisting Neutralizing Antibodies to Adeno-Associated Virus Capsids in Large Animals Other Than Monkeys May Confound In Vivo Gene Therapy Studies.

scientific article published in June 2015

Preexisting immunity to adenovirus in rhesus monkeys fails to prevent vector-induced toxicity

scientific article

Preparation of Nonhuman Primate Eyes for Histological Evaluation After Retinal Gene Transfer

scientific article published on 01 June 2018

Preparation of a DNA Standard for Quantitative Polymerase Chain Reaction (qPCR)

scientific article published on 01 August 2019

Production of recombinant adeno-associated virus

scientific article

Progress in the commercial-scale production of adeno-associated viral vectors

scientific article published on 01 July 2009

Prolonged correction of hyperlipidemia in mice with familial hypercholesterolemia using an adeno-associated viral vector expressing very-low-density lipoprotein receptor.

scientific article published in September 2000

Prolonged transgene expression in cotton rat lung with recombinant adenoviruses defective in E2a.

scientific article published in October 1994

Proteasome inhibition enhances AAV-mediated transgene expression in human synoviocytes in vitro and in vivo.

scientific article

Pseudotyped AAV vector-mediated gene transfer in a human fetal trachea xenograft model: implications for in utero gene therapy for cystic fibrosis

scientific article

Pseudotyped adeno-associated viral vector tropism and transduction efficiencies in murine wound healing

scientific article published on 19 June 2012

Pulmonary delivery of adenovirus vector formulated with dexamethasone-spermine facilitates homologous vector re-administration.

scientific article published on 27 September 2007

RPGR-associated retinal degeneration in human X-linked RP and a murine model

scientific article

Rapid, simple, and versatile manufacturing of recombinant adeno-associated viral vectors at scale.

scientific article published on October 2010

Recollections from a Pioneer Who Provided the Foundation for the Success of Gene Therapy in Treating Severe Combined Immune Deficiencies.

scientific article published in June 2016

Recombinant IL-12 prevents formation of blocking IgA antibodies to recombinant adenovirus and allows repeated gene therapy to mouse lung.

scientific article published in September 1995

Recombinant adeno-associated virus integration sites in murine liver after ornithine transcarbamylase gene correction

scientific article published on May 2013

Recombinant adeno-associated virus preferentially transduces human, compared to mouse, synovium: implications for arthritis therapy

scientific article published in January 2004

Recombinant adenovirus gene transfer in adults with partial ornithine transcarbamylase deficiency (OTCD).

scientific article published in September 1999

Regulated delivery of therapeutic proteins after in vivo somatic cell gene transfer.

scientific article published in January 1999

Regulated expression of erythropoietin from an AAV vector safely improves the anemia of beta-thalassemia in a mouse model.

scientific article published in April 2003

Regulated gene expression in gene therapy.

scientific article

Regulation of gene therapy in china

scientific article published in October 2006

Regulatory and Exhausted T Cell Responses to AAV Capsid.

scientific article published on 21 March 2017

Rep/Cap gene amplification and high-yield production of AAV in an A549 cell line expressing Rep/Cap.

scientific article

Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial.

scientific article

Rescue of chimeric adenoviral vectors to expand the serotype repertoire

scientific article published on 2 January 2007

Rescue of skeletal muscles of gamma-sarcoglycan-deficient mice with adeno-associated virus-mediated gene transfer.

scientific article published on February 2000

Resolution of primary severe acute respiratory syndrome-associated coronavirus infection requires Stat1.

scientific article published on October 2004

Rhesus monkey (Macaca mulatta) mucosal antimicrobial peptides are close homologues of human molecules

scientific article

Role of CFTR in lysosome acidification

scientific article published on April 15, 1992

Route of administration determines induction of T-cell-independent humoral responses to adeno-associated virus vectors.

scientific article published in April 2000

SARS vaccine: progress and challenge

scientific article

Safe and Sustained Expression of Human Iduronidase After Intrathecal Administration of Adeno-Associated Virus Serotype 9 in Infant Rhesus Monkeys

scientific article published on 10 June 2019

Safety and feasibility of liver-directed ex vivo gene therapy for homozygous familial hypercholesterolemia.

scientific article published in February 1996

Selective gene transfer into the liver of non-human primates with E1-deleted, E2A-defective, or E1-E4 deleted recombinant adenoviruses.

scientific article published on March 1998

Self-reactive CFTR T cells in humans: implications for gene therapy.

scientific article published on 19 July 2013

Sensitive Determination of Infectious Titer of Recombinant Adeno-Associated Viruses (rAAVs) Using TCID50 End-Point Dilution and Quantitative Polymerase Chain Reaction (qPCR)

scientific article published on 01 May 2020

Severe toxicity in nonhuman primates and piglets following high-dose intravenous administration of an AAV vector expressing human SMN.

scientific article published on 29 January 2018

Somatic gene transfer in the development of an animal model for primary hyperparathyroidism ⬇️

scientific article published on May 1, 1992

Specific AAV serotypes stably transduce primary hippocampal and cortical cultures with high efficiency and low toxicity

scientific article published on 17 November 2007

Stable liver-specific expression of human IDOL in humanized mice raises plasma cholesterol

scientific article published on 19 January 2016

Standardized Method for Intra-Cisterna Magna Delivery Under Fluoroscopic Guidance in Nonhuman Primates

scientific article published on 14 August 2018

Stat1-dependent induction of tumor necrosis factor-related apoptosis-inducing ligand and the cell-surface death signaling pathway by interferon beta in human cancer cells.

scientific article published on September 2003

Stroma formation and angiogenesis by overexpression of growth factors, cytokines, and proteolytic enzymes in human skin grafted to SCID mice

scientific article published in April 2003

Structure of neurotropic adeno-associated virus AAVrh.8.

scientific article

Structure-based identification of a major neutralizing site in an adenovirus hexon

scientific article published on 15 November 2006

Submucosal glands are the predominant site of CFTR expression in the human bronchus

scientific article published on 01 November 1992

Susceptibility to SIV Infection After Adenoviral Vaccination in a Low Dose Rhesus Macaque Challenge Model

Sustained correction of OTC deficiency in spf( ash) mice using optimized self-complementary AAV2/8 vectors.

scientific article published on 18 August 2011

Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy

scientific article published on 6 January 2005

Sustained production of beta-glucuronidase from localized sites after AAV vector gene transfer results in widespread distribution of enzyme and reversal of lysosomal storage lesions in a large volume of brain in mucopolysaccharidosis VII mice.

scientific article published in November 1999

Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

scientific article published on 12 August 2009

Systematic evaluation of AAV vectors for liver directed gene transfer in murine models.

scientific article

T helper cell-independent antibody responses to the transgene product of an e1-deleted adenoviral vaccine require NK1.1 T cells.

scientific article published on January 2003

TLR9 signaling mediates adaptive immunity following systemic AAV gene therapy

scientific article published on 26 October 2019

Tachi Yamada: An Academic, Drug Developer and Humanist

scientific article published on 01 December 2018

Tailoring the AAV vector capsid for gene therapy.

scientific article published on 04 December 2008

Targeted retroviral gene transfer into the rat biliary tract.

scientific article

Targeted transduction patterns in the mouse brain by lentivirus vectors pseudotyped with VSV, Ebola, Mokola, LCMV, or MuLV envelope proteins

scientific article published on May 2002

Targeting viral-mediated transduction to the lung airway epithelium with the anti-inflammatory cationic lipid dexamethasone-spermine.

scientific article published on September 2005

Temporary amelioration of hyperlipidemia in low density lipoprotein receptor-deficient rabbits transplanted with genetically modified hepatocytes

scientific article

The AAV9 receptor and its modification to improve in vivo lung gene transfer in mice

scientific article

The GPI-Linked Protein LY6A Drives AAV-PHP.B Transport across the Blood-Brain Barrier

scientific article published on 20 February 2019

The Gene Therapy Resource Program: A Decade of Dedication to Translational Research by the National Heart, Lung, and Blood Institute

scientific article published on 12 November 2017

The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice.

scientific article published on 2 February 2018

The Next Chapter

scientific article published on 01 June 2015

The Past, Present, and Future of Gene Therapy from Nobel Laureate David Baltimore

scientific article published on 22 May 2017

The RAC Retires After a Job Well Done

scientific article published on 01 September 2018

The Story of RNA Interference as a New Therapeutic Paradigm from Nobel Laureate Craig Mello

scientific article published in September 2017

The common variant of cystic fibrosis transmembrane conductance regulator is recognized by hsp70 and degraded in a pre-Golgi nonlysosomal compartment

scientific article published on October 1993

The complex and evolving story of T cell activation to AAV vector-encoded transgene products

scientific article

The first journal on human gene therapy celebrates its 25th anniversary.

scientific article published on January 2014

The innate immune system in cystic fibrosis lung disease

scientific article

The low density lipoprotein receptor is not required for normal catabolism of Lp(a) in humans

scientific article

The p38 mitogen-activated protein kinase signaling pathway is coupled to Toll-like receptor 5 to mediate gene regulation in response to Pseudomonas aeruginosa infection in human airway epithelial cells.

scientific article

The pleiotropic effects of natural AAV infections on liver-directed gene transfer in macaques

scientific article

The role of apoptosis in immune hyporesponsiveness following AAV8 liver gene transfer

scientific article published on 15 October 2013

The special case of gene therapy pricing

scientific article published in September 2014

The structure of AAVrh32.33, a novel gene delivery vector

scientific article

The technology of gene therapy.

scientific article published in February 2011

Therapeutic strategies for familial hypercholesterolemia based on somatic gene transfer.

scientific article

There and Back Again: Mitchell Finer on the Journey of Biotech from Start-Up to Success.

scientific article

Toll-like receptor 4 mediates innate immune responses to Haemophilus influenzae infection in mouse lung

scientific article

Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype.

scientific article published on 9 October 2003

Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Human Alpha-L-Iduronidase in Rhesus Macaques

scientific article published on 14 July 2018

Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Iduronate-2-Sulfatase in Rhesus Macaques

scientific article published on 14 July 2018

Transduction efficiencies of novel AAV vectors in mouse airway epithelium in vivo and human ciliated airway epithelium in vitro.

scientific article

Transduction of human islets with pseudotyped lentiviral vectors.

scientific article published on February 2004

Transduction of satellite cells after prenatal intramuscular administration of lentiviral vectors

scientific article published on January 2005

Transfer of a cathelicidin peptide antibiotic gene restores bacterial killing in a cystic fibrosis xenograft model

scientific article

Transfer of the CFTR Gene to the Lung of Nonhuman Primates with E1-Deleted, E2a-Defective Recombinant Adenoviruses: A Preclinical Toxicology Study ⬇️

scientific article published on July 1, 1995

Transient depletion of CD4 lymphocyte improves efficacy of repeated administration of recombinant adenovirus in the ornithine transcarbamylase deficient sparse fur mouse.

scientific article published in October 2000

Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases

scientific article published in February 2013

Translational Feasibility of Lumbar Puncture for Intrathecal AAV Administration

scientific article published on 18 April 2020

Transplantation of genetically modified autologous hepatocytes into nonhuman primates: feasibility and short-term toxicity.

scientific article

Treatment of advanced CNS malignancies with the recombinant adenovirus H5.010RSVTK: a phase I trial.

scientific article

Treatment of advanced mesothelioma with the recombinant adenovirus H5.010RSVTK: a phase 1 trial (BB-IND 6274)

scientific article published on 01 October 1996

Universal protection against influenza infection by a multidomain antibody to influenza hemagglutinin

scientific article

University Flunk-Out to Genomics Pioneer: An Interview with George Church, PhD

scientific article published on 14 September 2018

Upregulation of class I major histocompatibility complex antigens by interferon gamma is necessary for T-cell-mediated elimination of recombinant adenovirus-infected hepatocytes in vivo

scientific article

Use of chimeric adenoviral vectors to assess capsid neutralization determinants.

scientific article

Utility of PEGylated recombinant adeno-associated viruses for gene transfer.

scientific article published on 26 August 2005

Vaccines based on novel adeno-associated virus vectors elicit aberrant CD8+ T-cell responses in mice

scientific article published on 22 August 2007

Vector sequences are not detected in tumor tissue from research subjects with ornithine transcarbamylase deficiency who previously received adenovirus gene transfer

scientific article published on September 2013

Vectored expression of the broadly neutralizing antibody FI6 in mouse airway provides partial protection against a new avian influenza A virus, H7N9.

scientific article

Widespread dispersion of adeno-associated virus serotype 1 and adeno-associated virus serotype 6 vectors in the rat central nervous system and in human glioblastoma multiforme xenografts

scientific article

Widespread gene transfer in the central nervous system of cynomolgus macaques following delivery of AAV9 into the cisterna magna

scientific article published on 10 December 2014

Wnt10b and Dkk-1 gene therapy differentially influenced trabecular bone architecture, soft tissue integrity, and osteophytosis in a skeletally mature rat model of osteoarthritis.

scientific article

Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses.

scientific article

beta-Defensin 1 contributes to pulmonary innate immunity in mice

scientific journal article

α-Fetoprotein gene delivery to the nasal epithelium of nonhuman primates by human parainfluenza viral vectors

scientific article

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