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List of works by Donald B. Kohn

A CD19/Fc fusion protein for detection of anti-CD19 chimeric antigen receptors.

scientific article

A clinical trial of retroviral-mediated transfer of a rev-responsive element decoy gene into CD34(+) cells from the bone marrow of human immunodeficiency virus-1-infected children.

scientific article published in July 1999

A peptidyl derivative of [3H]aniline as a sensitive, stable, protease substrate

scientific article published on September 1979

A tale of two SCIDs

scientific article published in August 2011

Adenosine Deaminase (ADA)-Deficient Severe Combined Immune Deficiency (SCID) in the US Immunodeficiency Network (USIDNet) Registry

scientific article published on 02 September 2020

Adenosine deaminase gene therapy protocol revisited

scientific article published in February 2002

Adoptive transfer of MART-1 T-cell receptor transgenic lymphocytes and dendritic cell vaccination in patients with metastatic melanoma

scientific article

Advances in lentiviral vector design for gene-modification of hematopoietic stem cells.

scientific article

Allelic exclusion and peripheral reconstitution by TCR transgenic T cells arising from transduced human hematopoietic stem/progenitor cells

scientific article published on 5 February 2013

Allogeneic hematopoietic cell transplantation for primary immune deficiency diseases: current status and critical needs

scientific article

Amendment to Clinical Research Project. Project 90-C-195. April 1, 1993. Treatment of severe combined immunodeficiency disease (SCID) due to adenosine deaminase deficiency with autologous lymphocytes transduced with a human ADA gene.

scientific article published in February 1999

American Society of Gene Therapy (ASGT) ad hoc subcommittee on retroviral-mediated gene transfer to hematopoietic stem cells

scientific article

An in vitro model of human red blood cell production from hematopoietic progenitor cells.

scientific article

Analysis of optimal conditions for retroviral-mediated transduction of primitive human hematopoietic cells

scientific article

Antitumor activity from antigen-specific CD8 T cells generated in vivo from genetically engineered human hematopoietic stem cells.

scientific article published on 28 November 2011

Association of prostate-specific membrane antigen with caveolin-1 and its caveolae-dependent internalization in microvascular endothelial cells: implications for targeting to tumor vasculature

scientific article

Autologous bone marrow transplantation with 4-hydroperoxycyclophosphamide purged marrows for children with acute non-lymphoblastic leukemia in second remission

scientific article

B cell differentiation and IL-21 response in IL2RG/JAK3 SCID patients after hematopoietic stem cell transplantation.

scientific article

BTK mediated apoptosis, a possible mechanism for failure to generate high titer retroviral producer clones

scientific article published in May 2000

Bone marrow transplantation for genetic diseases

scientific article

Bone marrow transplantation for immunodeficiency and genetic diseases.

scientific article published on January 1990

Bone marrow transplantation for metabolic diseases.

scientific article

Bone marrow transplantation in primary disorders of the haematopoietic stem cell

scientific article published on December 1989

Busulfan and cyclophosphamide as a conditioning regimen for pediatric acute lymphoblastic leukemia patients undergoing bone marrow transplantation

scientific article published in February 2004

CARs on track in the clinic

scientific article

CD4+CD25- T cells transduced to express MHC class I-restricted epitope-specific TCR synthesize Th1 cytokines and exhibit MHC class I-restricted cytolytic effector function in a human melanoma model

scientific article published on July 2008

Carrier detection in the Wiskott Aldrich syndrome

scientific article

Cells expressing human glucocerebrosidase from a retroviral vector repopulate macrophages and central nervous system microglia after murine bone marrow transplantation

scientific article published in May 1994

Challenges in vector and trial design using retroviral vectors for long-term gene correction in hematopoietic stem cell gene therapy

scientific article published in June 2012

Chemistry, manufacturing and controls for gene modified hematopoietic stem cells

scientific article published on 08 February 2019

Chronic Granulomatous Disease-Associated IBD Resolves and Does Not Adversely Impact Survival Following Allogeneic HCT

scientific article published on 02 August 2019

Clinical and genetic heterogeneity in Omenn syndrome and severe combined immune deficiency

scientific article published on 05 September 2008

Clinical applications of gene therapy

scientific article published on 01 August 2000

Clonality analysis after retroviral-mediated gene transfer to CD34+ cells from the cord blood of ADA-deficient SCID neonates

scientific article published on 17 March 2003

Combination of CD80 and granulocyte-macrophage colony-stimulating factor coexpression by a leukemia cell vaccine: preclinical studies in a murine model recapitulating Philadelphia chromosome-positive acute lymphoblastic leukemia

scientific article published on September 1999

Comparison of gene transfer efficiencies and gene expression levels achieved with equine infectious anemia virus- and human immunodeficiency virus type 1-derived lentivirus vectors

scientific article

Comparison of the effects of growth factors on retroviral vector-mediated gene transfer and the proliferative status of human hematopoietic progenitor cells

scientific article

Comparison of trans-dominant inhibitory mutant human immunodeficiency virus type 1 genes expressed by retroviral vectors in human T lymphocytes.

scientific article published on June 1993

Consensus approach for the management of severe combined immune deficiency caused by adenosine deaminase deficiency

scientific article published on 05 September 2018

Constitutive HOXA5 expression inhibits erythropoiesis and increases myelopoiesis from human hematopoietic progenitors

scientific article

Correction of adenosine deaminase deficiency in cultured human T and B cells by retrovirus-mediated gene transfer

scientific article published on September 1986

Correction of interleukin-2 receptor function in X-SCID lymphoblastoid cells by retrovirally mediated transfer of the gamma-c gene

scientific article published on April 1996

Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells

scientific article published on 2 March 2015

Correction to: Infections in Infants with SCID: Isolation, Infection Screening and Prophylaxis in PIDTC Centers

scientific article published on 03 December 2020

Correction: Chronic Granulomatous Disease-Associated IBD Resolves and Does Not Adversely Impact Survival Following Allogeneic HCT

scientific article published on 29 August 2020

Critical factors influencing stable transduction of human CD34(+) cells with HIV-1-derived lentiviral vectors

scientific article published on July 2000

Direct FGF-2 gene transfer via recombinant adeno-associated virus vectors stimulates cell proliferation, collagen production, and the repair of experimental lesions in the human ACL.

scientific article published on 19 November 2012

Dissecting the mechanism of histone deacetylase inhibitors to enhance the activity of zinc finger nucleases delivered by integrase-defective lentiviral vectors.

scientific article

Dynamic tracking of human hematopoietic stem cell engraftment using in vivo bioluminescence imaging

scientific article

Effective suicide gene therapy for leukemia in a model of insertional oncogenesis in mice

scientific article published on January 2007

Effects of busulfan dose escalation on engraftment of infant rhesus monkey hematopoietic stem cells after gene marking by a lentiviral vector

scientific article

Effects of the negative control region on expression from retroviral LTR

scientific article published on 01 April 2003

Effects of vector backbone and pseudotype on lentiviral vector-mediated gene transfer: studies in infant ADA-deficient mice and rhesus monkeys

scientific article published on 13 June 2014

Efficient characterization of retro-, lenti-, and foamyvector-transduced cell populations by high-accuracy insertion site sequencing.

scientific article published in May 2003

Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency

scientific article

Erythropoiesis from human embryonic stem cells through erythropoietin-independent AKT signaling

scientific article

Establishing diagnostic criteria for severe combined immunodeficiency disease (SCID), leaky SCID, and Omenn syndrome: the Primary Immune Deficiency Treatment Consortium experience

scientific article

Establishment and characterization of adenosine deaminase-deficient human T cell lines.

scientific article published in June 1989

Ethical and regulatory aspects of genome editing.

scientific article published on 6 April 2016

Excellent Outcomes Following Hematopoietic Cell Transplantation for Wiskott-Aldrich Syndrome: A PIDTC Report

scientific article published on 08 April 2020

Expansion of multipotent and lymphoid-committed human progenitors through intracellular dimerization of Mpl.

scientific article published on 3 January 2008

Expression from second-generation feline immunodeficiency virus vectors is impaired in human hematopoietic cells

scientific article published in November 2002

Expression levels by retroviral vectors based upon the N2 and the MFG backbones

scientific article published in April 1996

Expression of biologically active human factor IX in human hematopoietic cells after retroviral vector-mediated gene transduction

scientific article published in July 1995

Expression of coagulation factor IX (Christmas factor) in human hepatoma (HepG2) cell cultures after retroviral vector-mediated transfer

scientific article published on 01 May 1993

Expression of hammerhead ribozymes by retroviral vectors to inhibit HIV-1 replication: comparison of RNA levels and viral inhibition

scientific article published in January 1996

Expression of human Wiskott-Aldrich syndrome protein in patients' cells leads to partial correction of a phenotypic abnormality of cell surface glycoproteins

scientific article

Expression of human adenosine deaminase in nonhuman primates after retrovirus-mediated gene transfer

scientific article

Expression of human glucocerebrosidase following retroviral vector-mediated transduction of murine hematopoietic stem cells

scientific article published in November 1991

Expression of human glucocerebrosidase in murine long-term bone marrow cultures after retroviral vector-mediated transfer.

scientific article published on February 1990

FLT3 ligand preserves the ability of human CD34+ progenitors to sustain long-term hematopoiesis in immune-deficient mice after ex vivo retroviral-mediated transduction

scientific article published on 01 January 1997

Factors influencing the titer and infectivity of lentiviral vectors.

scientific article

Fetal gene transfer using lentiviral vectors and the potential for germ cell transduction in rhesus monkeys (Macaca mulatta).

scientific article

Foamy virus vectors expressing anti-HIV transgenes efficiently block HIV-1 replication.

scientific article published on 23 October 2007

From skin biopsy to neurons through a pluripotent intermediate under Good Manufacturing Practice protocols

scientific article published on 07 December 2011

Gene delivery to human B-precursor acute lymphoblastic leukemia cells.

scientific article

Gene therapy comes of age.

scientific article published in January 2018

Gene therapy for HIV-1 infection.

scientific article published on January 1996

Gene therapy for XSCID: the first success of gene therapy

scientific article published in November 2000

Gene therapy for adenosine deaminase deficiency

scientific article

Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans.

scientific article

Gene therapy for blood diseases

scientific article published on 29 December 2018

Gene therapy for childhood immunological diseases

scientific article published on 12 November 2007

Gene therapy for genetic diseases

scientific article

Gene therapy for genetic haematological disorders and immunodeficiencies

scientific article

Gene therapy for haematopoietic and lymphoid disorders.

scientific article published on January 1997

Gene therapy for hematopoietic and immune disorders

scientific article published on December 1996

Gene therapy for inborn and acquired immune deficiency disorders

scientific article published on January 2003

Gene therapy for newborns.

scientific article

Gene therapy for pediatric AIDS.

scientific article

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

scientific article (publication date: April 2013)

Gene therapy for the treatment of recurrent pediatric malignant astrocytomas with in vivo tumor transduction with the herpes simplex thymidine kinase gene/ganciclovir system

scientific article published on July 1994

Gene therapy fulfilling its promise

scientific article

Gene therapy of RAG-2-/- mice: sustained correction of the immunodeficiency

scientific article published on 22 August 2002

Gene therapy outpaces haplo for SCID-X1.

scientific article published in June 2015

Gene therapy through autologous transplantation of gene-modified hematopoietic stem cells

scientific article

Gene therapy to inhibit xenoantibody production using lentiviral vectors in non-human primates

scientific article published on 3 August 2006

Gene therapy using hematopoietic stem cells: Sisyphus approaches the crest

scientific article

Gene therapy/bone marrow transplantation in ADA-deficient mice: roles of enzyme-replacement therapy and cytoreduction

scientific article published on 25 July 2012

Gene therapy: WAS (not) just for kids

scientific article published in September 2017

Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013.

scientific article

Gene therapy: efforts at developing large animal models for autologous bone marrow transplant and gene transfer with retroviral vectors

scientific article

Gene transfer and expression in nonhuman primates using retroviral vectors

scientific article

Gene transfer into human umbilical cord blood-derived CD34+ cells by particle-mediated gene transfer

scientific article published in May 1998

Generation and characterization of transgene-free human induced pluripotent stem cells and conversion to putative clinical-grade status

scientific article

Genetic therapies against HIV.

scientific article published on December 2007

Genetically corrected autologous stem cells engraft, but host immune responses limit their utility in canine alpha-L-iduronidase deficiency

scientific article published on 01 March 1999

Granulocyte recovery in pediatric marrow transplant recipients treated with ganciclovir for cytomegalovirus infection.

scientific article

Growth factors increase amphotropic retrovirus binding to human CD34+ bone marrow progenitor cells

scientific article published in December 1993

Guidance for developing phase II cell therapy trial proposals for consideration by the Blood and Marrow Transplant Clinical Trials Network

scientific article

HIV eradication--from Berlin to Boston

scientific article

HIV-1-derived lentiviral vectors and fetal route of administration on transgene biodistribution and expression in rhesus monkeys

scientific article published in May 2005

Hematopoietic Cell Transplantation in Patients With Primary Immune Regulatory Disorders (PIRD): A Primary Immune Deficiency Treatment Consortium (PIDTC) Survey

scientific article published on 21 February 2020

Hematopoietic stem cell gene therapy for the multisystemic lysosomal storage disorder cystinosis

scientific article published on 23 October 2012

Hematopoietic stem cell transplantation for primary lymphoid immunodeficiencies

scientific article published on 01 October 1998

Hematopoietic stem cell transplantation for severe combined immune deficiency

scientific article

Hematopoietic stem cells for cancer immunotherapy

scientific article published on January 2014

High-resolution analysis of cytosine methylation in the 5ĺong terminal repeat of retroviral vectors

scientific article

Highly efficient large-scale lentiviral vector concentration by tandem tangential flow filtration

scientific article

Human Hematopoietic Cell Culture, Transduction, and Analyses

article

Human gene marker/therapy clinical protocols

scientific article published on 01 April 2000

Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo

scientific article

IND-enabling studies for a clinical trial to genetically program a persistent cancer-targeted immune system

scientific article published on 08 November 2018

Immune reconstitution and survival of 100 SCID patients post-hematopoietic cell transplant: a PIDTC natural history study.

scientific article published on 11 October 2017

Immune response to green fluorescent protein: implications for gene therapy

scientific article published in July 1999

Immunologic studies of lymph node lymphocytes in the generalized lymphadenopathy syndrome

scientific article published in January 1987

Immunotherapy against murine leukemia.

scientific article

Improved expression in hematopoietic and lymphoid cells in mice after transplantation of bone marrow transduced with a modified retroviral vector

scientific article published on November 1999

Improving cellular therapy for primary immune deficiency diseases: recognition, diagnosis, and management

scientific article

In Vivo Transduction by Intravenous Injection of a Lentiviral Vector Expressing Human ADA into Neonatal ADA Gene Knockout Mice: A Novel Form of Enzyme Replacement Therapy for ADA Deficiency

article

In utero injection of alpha-L-iduronidase-carrying retrovirus in canine mucopolysaccharidosis type I: infection of multiple tissues and neonatal gene expression

scientific article published in October 2002

In vivo biosafety model to assess the risk of adverse events from retroviral and lentiviral vectors

scientific article

Increased gene transfer into human CD34+ progenitor cells using retroviral vectors produced by a canine packaging cell line

scientific article published on January 1998

Increased levels of spliced RNA account for augmented expression from the MFG retroviral vector in hematopoietic cells

scientific article

Increased probability of expression from modified retroviral vectors in embryonal stem cells and embryonal carcinoma cells

scientific article published on December 1997

Infection of human hematopoietic progenitor cells using a retroviral vector with a xenotropic pseudotype.

scientific article

Infection of human marrow stroma by human immunodeficiency virus-1 (HIV-1) is both required and sufficient for HIV-1-induced hematopoietic suppression in vitro: demonstration by gene modification of primary human stroma.

scientific article

Infections in Infants with SCID: Isolation, Infection Screening, and Prophylaxis in PIDTC Centers

scientific article published on 02 October 2020

Inhibition of HIV-1 replication using a mutated tRNALys-3 primer

scientific article published on June 1997

Inhibition of human immunodeficiency virus-1 (HIV-1) replication after transduction of granulocyte colony-stimulating factor-mobilized CD34+ cells from HIV-1-infected donors using retroviral vectors containing anti-HIV-1 genes.

scientific article published in April 1997

Integrase-defective lentiviral vectors as a delivery platform for targeted modification of adenosine deaminase locus

scientific article published on 16 July 2013

Integrated self-inactivating lentiviral vectors produce full-length genomic transcripts competent for encapsidation and integration

scientific article published on August 2004

Intrakines--evidence for a trans-cellular mechanism of action

scientific article published on February 2000

Intrapulmonary and intramyocardial gene transfer in rhesus monkeys (Macaca mulatta): safety and efficiency of HIV-1-derived lentiviral vectors for fetal gene delivery

scientific article published in July 2005

Kinetics of fluorescence expression in nonhuman primates transplanted with GFP retrovirus-modified CD34 cells

scientific article published on 01 July 2002

Lack of expression from a retroviral vector after transduction of murine hematopoietic stem cells is associated with methylation in vivo

scientific article

Lentiviral gene therapy for X-linked chronic granulomatous disease

scientific article published on 27 January 2020

Lentiviral vector gene transfer into fetal rhesus monkeys (Macaca mulatta): lung-targeting approaches

scientific article published in December 2001

Lentiviral vector transduction of a dominant-negative Rev gene into human CD34+ hematopoietic progenitor cells potently inhibits human immunodeficiency virus-1 replication

scientific article published on January 2007

Lentiviral vectors for efficient delivery of CD80 and granulocyte-macrophage- colony-stimulating factor in human acute lymphoblastic leukemia and acute myeloid leukemia cells to induce antileukemic immune responses

scientific article published on August 2000

Lentiviral vectors ready for prime-time

scientific article published on 01 January 2007

Lentiviral vectors with amplified beta cell-specific gene expression

scientific article published on 14 May 2009

Lentivirus Mediated Correction of Artemis-Deficient Severe Combined Immunodeficiency

scientific article published on 9 September 2016

Lentivirus vectors incorporating the immunoglobulin heavy chain enhancer and matrix attachment regions provide position-independent expression in B lymphocytes

scientific article

Letter to the editors of Nature from the American Society of Gene Therapy (ASGT) and the European Society of Gene Therapy (ESGT).

scientific article published in July 2003

Long-term efficacy of enzyme replacement therapy for adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID).

scientific article published on 22 August 2005

Long-term in vivo monitoring of mouse and human hematopoietic stem cell engraftment with a human positron emission tomography reporter gene

scientific article published on 14 January 2013

Long-term neurocognitive function of pediatric patients with severe combined immune deficiency (SCID): pre- and post-hematopoietic stem cell transplant (HSCT).

scientific article published on 20 September 2008

Macrophages can recognize and kill tumor cells bearing the membrane isoform of macrophage colony-stimulating factor.

scientific article published in June 1996

Manipulation of OCT4 levels in human embryonic stem cells results in induction of differential cell types

scientific article published on November 2007

Marrow-derived cells as vehicles for delivery of gene therapy to pulmonary epithelium

scientific article published in December 2002

Modification of hematopoietic stem/progenitor cells with CD19-specific chimeric antigen receptors as a novel approach for cancer immunotherapy.

scientific article published on October 2013

Morphological analysis and lentiviral transduction of fetal monkey bone marrow-derived mesenchymal stem cells

scientific article published in January 2004

Multiple modifications in cis elements of the long terminal repeat of retroviral vectors lead to increased expression and decreased DNA methylation in embryonic carcinoma cells.

scientific article published in February 1995

Mycobacterium avium-intracellulare infections after allogeneic bone marrow transplantation in children.

scientific article published in January 1990

Myeloid dysplasia and bone marrow hypocellularity in adenosine deaminase-deficient severe combined immune deficiency.

scientific article

Myoblast gene therapy in canine mucopolysaccharidosis. I: Abrogation by an immune response to alpha-L-iduronidase

scientific article

Neonatal bone marrow transplantation of ADA-deficient SCID mice results in immunologic reconstitution despite low levels of engraftment and an absence of selective donor T lymphoid expansion

scientific article published on 20 March 2008

Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector

scientific article

Neurocognitive function of patients with severe combined immunodeficiency

scientific article published in February 2010

Newborn Screening for Severe Combined Immunodeficiency and T-cell Lymphopenia in California, 2010-2017

scientific article published on 01 February 2019

Newborn screening for severe combined immunodeficiency and T-cell lymphopenia in California: results of the first 2 years

scientific article published on July 2013

Newborn screening for severe combined immunodeficiency in 11 screening programs in the United States

scientific article published in August 2014

Nonmyeloablative conditioning regimen to increase engraftment of gene-modified hematopoietic stem cells in young rhesus monkeys

scientific article

Novel Pol II fusion promoter directs human immunodeficiency virus type 1-inducible coexpression of a short hairpin RNA and protein.

scientific article

Novel pathways to erythropoiesis induced by dimerization of intracellular C-Mpl in human hematopoietic progenitors

scientific article published on April 2012

Occurrence of leukaemia following gene therapy of X-linked SCID.

scientific article

Overexpression of tissue inhibitor of metalloproteinases-2 retroviral-mediated gene transfer in vivo inhibits tumor growth and invasion

scientific article

Perspectives on gene therapy for immune deficiencies

scientific article published in December 2005

Potential applications of gene therapy

scientific article published on November 1989

Pre- and post-natal treatment of hemophagocytic lymphohistiocytosis

scientific article published on January 2009

Preclinical correction of human Fanconi anemia complementation group A bone marrow cells using a safety-modified lentiviral vector

scientific article

Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency

scientific article

Preferential association of prostate cancer cells expressing prostate specific membrane antigen to bone marrow matrix.

scientific article published in April 2007

Preloading potential of retroviral vectors is packaging cell clone dependent and centrifugation onto CH-296 ensures highest transduction efficiency.

scientific article published on April 2009

Primary Immune Deficiency Treatment Consortium (PIDTC) report

scientific article published on 15 October 2013

Primary Immune Deficiency Treatment Consortium (PIDTC) update

scientific article published on 22 April 2016

Progressive declines in neurocognitive function among survivors of hematopoietic stem cell transplantation for pediatric hematologic malignancies

scientific article published on 01 June 2008

Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism: a case report

scientific article published on 14 September 2006

Prostate cancer originating in basal cells progresses to adenocarcinoma propagated by luminal-like cells

scientific article published on 26 November 2013

Recombinant adeno-associated virus mediates a high level of gene transfer but less efficient integration in the K562 human hematopoietic cell line

scientific article

Recombinant murine interleukin-12 elicits potent antileukemic immune responses in a murine model of Philadelphia chromosome-positive acute lymphoblastic leukemia

scientific article

Reconstitution of T cell receptor signaling in ZAP-70-deficient cells by retroviral transduction of the ZAP-70 gene.

scientific article

Redirecting specificity of T-cell populations for CD19 using the Sleeping Beauty system

scientific article published on April 2008

Regulated expansion of human pancreatic beta-cells

scientific article

Requirement for NK cells in CD40 ligand-mediated rejection of Philadelphia chromosome-positive acute lymphoblastic leukemia cells

scientific article

Rescue of splicing-mediated intron loss maximizes expression in lentiviral vectors containing the human ubiquitin C promoter

scientific article

Retroviral mediated transfer of the cDNA for human glucocerebrosidase into hematopoietic stem cells of patients with Gaucher disease. A phase I study

scientific article published on 01 January 1996

Retroviral transfer of the glucocerebrosidase gene into CD34+ cells from patients with Gaucher disease: in vivo detection of transduced cells without myeloablation

scientific article

Retroviral vector-mediated gene transfer into primitive human hematopoietic progenitor cells: effects of mast cell growth factor (MGF) combined with other cytokines

scientific article published in October 1992

Retroviral-mediated gene expression in human myelomonocytic cells: a comparison of hematopoietic cell promoters to viral promoters

scientific article published in October 1995

Retroviral-mediated gene transfer into hematopoietic cells.

scientific article published in January 1986

Retroviral-mediated gene transfer into hemopoietic cells

scientific article published on January 1988

Retroviral-mediated gene transfer into mammalian cells

scientific article published on January 1987

Retroviral-mediated transfer of the human glucocerebrosidase gene into cultured Gaucher bone marrow

scientific article published on August 1992

Retrovirally transduced bone marrow has a therapeutic effect on brain in the mouse model of mucopolysaccharidosis IIIB.

scientific article published in August 2004

Retrovirus-mediated transfer of the human alpha-L-iduronidase cDNA into human hematopoietic progenitor cells leads to correction in trans of Hurler fibroblasts

scientific article

Rhesus monkey model for fetal gene transfer: studies with retroviral- based vector systems

scientific article

SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery

scientific article published on 28 August 2018

Scaffold attachment region-containing retrovirus vectors improve long-term proviral expression after transplantation of GFP-modified CD34+ baboon repopulating cells

scientific article published on 17 July 2003

Second hematopoietic stem cell transplantation in pediatric patients: overall survival and long-term follow-up

scientific article published on 01 January 2002

Selective survival of peripheral blood lymphocytes in children with HIV-1 following delivery of an anti-HIV gene to bone marrow CD34(+) cells

scientific article

Short-course amphotericin B therapy for isolated candiduria in children

scientific article published on 01 February 1987

Simian immunodeficiency virus infection of hematopoietic stem cells and bone marrow stromal cells

scientific article published in May 2004

Simultaneous use of two retroviral vectors in human gene marking trials: feasibility and potential applications

scientific article

Specific and stable gene transfer to human embryonic stem cells using pseudotyped lentiviral vectors

scientific article

Stable gene transfer to human CD34(+) hematopoietic cells using the Sleeping Beauty transposon

scientific article published on October 2006

Stable transduction of quiescent CD34(+)CD38(-) human hematopoietic cells by HIV-1-based lentiviral vectors

scientific article

Stable transgene expression in primitive human CD34+ hematopoietic stem/progenitor cells, using the Sleeping Beauty transposon system

scientific article

Stem cell directed gene therapy

scientific article

Successful hematopoietic stem cell transplantation for Niemann-Pick disease type B.

scientific article

Suitability of bone marrow from HIV-1-infected donors for retrovirus-mediated gene transfer

scientific article published on February 1997

Superior lentiviral vectors designed for BSL-0 environment abolish vector mobilization

scientific article published on 06 September 2018

Supramolecular nanosubstrate-mediated delivery system enables CRISPR-Cas9 knockin of hemoglobin beta gene for hemoglobinopathies

scientific article published on 23 October 2020

Sustained human hematopoiesis in immunodeficient mice by cotransplantation of marrow stroma expressing human interleukin-3: analysis of gene transduction of long-lived progenitors

scientific article published on 01 May 1994

T cell depleted haploidentical bone marrow transplantation for the treatment of children with severe combined immunodeficiency.

scientific article published on January 2000

T lymphocyte ontogeny in adenosine deaminase-deficient severe combined immune deficiency after treatment with polyethylene glycol-modified adenosine deaminase

scientific article

T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates.

scientific article published on July 1998

The Moloney murine leukemia virus repressor binding site represses expression in murine and human hematopoietic stem cells

scientific article published on September 2003

The current status of gene therapy using hematopoietic stem cells.

scientific article published on February 1995

The effects of Campath 1H upon graft-versus-host disease, infection, relapse, and immune reconstitution in recipients of pediatric unrelated transplants

scientific article published on 23 March 2007

The enhanced green fluorescent protein (eGFP) is minimally immunogenic in C57BL/6 mice

scientific article

The expression cassette determines the functional activity of ribozymes in mammalian cells by controlling their intracellular localization.

scientific article

The genetic landscape of severe combined immunodeficiency in the United States and Canada in the current era (2010-2018)

scientific article published on 05 September 2018

The natural history of children with severe combined immunodeficiency: baseline features of the first fifty patients of the primary immune deficiency treatment consortium prospective study 6901.

scientific article published on 02 July 2013

The presence of an autologous marrow stromal cell layer increases glucocerebrosidase gene transduction of long-term culture initiating cells (LTCICs) from the bone marrow of a patient with Gaucher disease.

scientific article published in October 1995

The woodchuck hepatitis virus post-transcriptional regulatory element reduces readthrough transcription from retroviral vectors

scientific article

Tissue-specific restriction of cyclophilin A-independent HIV-1- and SIV-derived lentiviral vectors

scientific article

Tolerance induction by lentiviral gene therapy with a nonmyeloablative regimen

scientific article published on 15 November 2005

Toward gene therapy for Gaucher disease

scientific article published on January 1991

Tracheal aspirate examination for Pneumocystis carinii cysts as a guide to therapy in pneumocystis pneumonia

scientific article published on 01 June 1983

Transduction of green fluorescent protein increased oxidative stress and enhanced sensitivity to cytotoxic drugs in neuroblastoma cell lines

scientific article published in September 2003

Transduction of human CD34+ hematopoietic progenitor cells by a retroviral vector expressing an RRE decoy inhibits human immunodeficiency virus type 1 replication in myelomonocytic cells produced in long-term culture

scientific article published in July 1996

Transduction of human melanoma cell lines with the human interleukin-7 gene using retroviral-mediated gene transfer: comparison of immunologic properties with interleukin-2.

scientific article published on December 1993

Transduction of pluripotent human hematopoietic stem cells demonstrated by clonal analysis after engraftment in immune-deficient mice

scientific article published on March 1996

Transient gene expression by nonintegrating lentiviral vectors

scientific article published on 23 March 2006

Transplantation outcomes for severe combined immunodeficiency, 2000-2009.

scientific article

Treatment of the mouse model of mucopolysaccharidosis I with retrovirally transduced bone marrow

scientific article published in August 2003

Two different genes coding for processable and nonprocessable forms of a viral envelope protein can account for the apparent hormonal stimulation of protein processing in W7MG1 lymphoma cells

scientific article published on March 1992

Umbilical cord blood cell transduction by retroviral vectors: preclinical studies to optimize gene transfer

scientific article published on January 1994

Unrelated donor BMT for Wiskott-Aldrich syndrome

scientific article published on 01 August 1993

Update on gene therapy for immunodeficiencies

scientific article

Update on gene therapy of inherited immune deficiencies

scientific article published in October 2003

Use of a retroviral vector with an internal opsin promoter to direct gene expression to retinal photoreceptor cells

scientific article published on August 1996

Use of lentiviral vectors to induce long-term tolerance to gal(+) heart grafts

scientific article published in June 2004

[Transplantation of genetically modified cells in the treatment of children with SCID: great hopes and recent disappointments]

scientific article published on 01 January 2003

β-globin gene transfer to human bone marrow for sickle cell disease.

scientific article

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