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List of works by Jakub Tolar

3D Printed Functional and Biological Materials on Moving Freeform Surfaces.

scientific article

A 3D in vitro model of the dermoepidermal junction amenable to mechanical testing

scientific article published on 12 September 2018

A Functional Bioluminescent Zebrafish Screen for Enhancing Hematopoietic Cell Homing

scientific article published on 21 December 2016

A biologic Velcro patch

scientific article

A homozygous frameshift variant in the KRT5 gene is compatible with life and results in severe recessive epidermolysis bullosa simplex

scientific article published on 26 June 2019

A multitask clustering approach for single-cell RNA-seq analysis in Recessive Dystrophic Epidermolysis Bullosa

scientific article published on 9 April 2018

A novel explanation of corneal clouding in a bone marrow transplant-treated patient with Hurler syndrome

scientific article published on 25 May 2016

A novel method for KIR-ligand typing by pyrosequencing to predict NK cell alloreactivity

scientific article

A picornaviral 2A-like sequence-based tricistronic vector allowing for high-level therapeutic gene expression coupled to a dual-reporter system

scientific article published on September 2005

ATXN1-CIC Complex Is the Primary Driver of Cerebellar Pathology in Spinocerebellar Ataxia Type 1 through a Gain-of-Function Mechanism.

scientific article published on 7 March 2018

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

Acid sphingomyelinase deficiency does not protect from graft-versus-host disease in transplant recipients with Niemann-Pick disease

scientific article published on 01 January 2010

Adeno-associated virus-mediated delivery of a mutant endostatin in combination with carboplatin treatment inhibits orthotopic growth of ovarian cancer and improves long-term survival

scientific article published on April 2006

Advances in understanding and treating dystrophic epidermolysis bullosa

scientific article

Allogeneic blood and bone marrow cells for the treatment of severe epidermolysis bullosa: repair of the extracellular matrix

scientific article published on October 2013

Allogeneic hematopoietic SCT for alpha-mannosidosis: an analysis of 17 patients

scientific article published on 9 May 2011

Allogeneic hematopoietic cell transplantation in children with relapsed acute lymphoblastic leukemia isolated to the central nervous system

scientific article published on 01 June 2008

Alternative donor transplant of benign primary hematologic disorders

scientific article

Amelioration of epidermolysis bullosa by transfer of wild-type bone marrow cells

scientific article published on 27 October 2008

An exploratory analysis of mitochondrial haplotypes and allogeneic hematopoietic cell transplantation outcomes

scientific article

An exploratory study of brain function and structure in mucopolysaccharidosis type I: long term observations following hematopoietic cell transplantation (HCT)

scientific article

An irradiated marrow niche reveals a small non-collagenous protein mediator of homing, dermatopontin

scientific article published on 27 August 2021

Analysis of glycosaminoglycans in cerebrospinal fluid from patients with mucopolysaccharidoses by isotope-dilution ultra-performance liquid chromatography-tandem mass spectrometry

scientific article published on 16 May 2011

Angiotensin receptor blockade mediated amelioration of mucopolysaccharidosis type I cardiac and craniofacial pathology

scientific article published on 14 October 2016

Augmentation of cutaneous wound healing by pharmacologic mobilization of endogenous bone marrow stem cells

scientific article published on 01 September 2014

Bacterial meningitis from Rothia mucilaginosa in patients with malignancy or undergoing hematopoietic stem cell transplantation

scientific article published on 01 March 2008

Base Editor Correction of COL7A1 in Recessive Dystrophic Epidermolysis Bullosa Patient-Derived Fibroblasts and iPSCs

scientific article published on 19 August 2019

Bone marrow myeloid-derived suppressor cells (MDSCs) inhibit graft-versus-host disease (GVHD) via an arginase-1-dependent mechanism that is up-regulated by interleukin-13

scientific article

Bone marrow stromal and vascular smooth muscle cells have chemosensory capacity via bitter taste receptor expression

scientific article

Bone marrow transplant with post-transplant cyclophosphamide for recessive dystrophic epidermolysis bullosa expands the related donor pool and permits tolerance of nonhaematopoietic cellular grafts

scientific article published on 28 June 2019

Bone marrow transplantation for recessive dystrophic epidermolysis bullosa

scientific article

CRISPR/Cas9 Targeted Gene Editing and Cellular Engineering in Fanconi Anemia.

scientific article published on 18 August 2016

CRISPR/Cas9-Based Cellular Engineering for Targeted Gene Overexpression.

scientific article published on 22 March 2018

CRISPR/Cas9-Mediated Correction of the FANCD1 Gene in Primary Patient Cells

scientific article

CRISPR/Cas9-based genetic correction for recessive dystrophic epidermolysis bullosa

scientific article published on 8 December 2016

Cardiac Ultrasound Findings in Infants with Severe (Hurler Phenotype) Untreated Mucopolysaccharidosis (MPS) Type I ⬇️

scientific article published on February 12, 2013

Cardiac disease in mucopolysaccharidosis type I attributed to catecholaminergic and hemodynamic deficiencies

scientific article

Cardiac functional and histopathologic findings in humans and mice with mucopolysaccharidosis type I: implications for assessment of therapeutic interventions in hurler syndrome

scientific article published on 02 December 2005

Case 4-2008: A pregnant woman with a swollen left breast and dyspnea

scientific article published on 01 June 2008

Cerebral Spinal Fluid levels of Cytokines are elevated in Patients with Metachromatic Leukodystrophy

scientific article published on 15 April 2016

Cerebrospinal fluid matrix metalloproteinases are elevated in cerebral adrenoleukodystrophy and correlate with MRI severity and neurologic dysfunction

scientific article

Characterization and management of hypercalcemia following transplantation for osteopetrosis

scientific article published on 5 October 2009

Childhood cerebral X-linked adrenoleukodystrophy: diffusion tensor imaging measurements for prediction of clinical outcome after hematopoietic stem cell transplantation.

scientific article

Chimerism and tolerance in a recipient of a deceased-donor liver transplant

scientific article published on 01 May 2008

Chitotriosidase as a biomarker of cerebral adrenoleukodystrophy

scientific article published on 20 October 2011

Circulating anticoagulant glycosaminoglycans in mucopolysaccharidosis type I

scientific article published on 25 February 2008

Clear cells in the atrioventricular valves of infants with severe human mucopolysaccharidosis (Hurler syndrome) are activated valvular interstitial cells

scientific article published on 09 July 2010

Clinical trial of laronidase in Hurler syndrome after hematopoietic cell transplantation

scientific article published on 21 August 2019

Combination of enzyme replacement and hematopoietic stem cell transplantation as therapy for Hurler syndrome

scientific article published on 26 November 2007

Concise review: Transplantation of human hematopoietic cells for extracellular matrix protein deficiency in epidermolysis bullosa

scientific article published on June 2011

Concise review: hitting the right spot with mesenchymal stromal cells

scientific article published on August 2010

Correction of DNA protein kinase deficiency by spliceosome-mediated RNA trans-splicing and sleeping beauty transposon delivery

scientific article published on 24 April 2007

Correction: Rapid DNA replication origin licensing protects stem cell pluripotency.

scientific article published on 5 June 2019

Cross platform analysis of methylation, miRNA and stem cell gene expression data in germ cell tumors highlights characteristic differences by tumor histology.

scientific article

Current Knowledge and Priorities for Future Research in Late Effects after Hematopoietic Cell Transplantation for Inherited Bone Marrow Failure Syndromes: Consensus Statement from the Second Pediatric Blood and Marrow Transplant Consortium Internati

scientific article

Current international perspectives on hematopoietic stem cell transplantation for inherited metabolic disorders.

scientific article published on February 2010

Cyclophosphamide conditioning in patients with severe aplastic anaemia given unrelated marrow transplantation: a phase 1-2 dose de-escalation study

scientific article published on 2 September 2015

DNA methylation of Runx1 regulatory regions correlates with transition from primitive to definitive hematopoietic potential in vitro and in vivo

scientific article published on 12 September 2013

Depletion of endogenous tumor-associated regulatory T cells improves the efficacy of adoptive cytotoxic T-cell immunotherapy in murine acute myeloid leukemia

scientific article published on September 2009

Derivation and high engraftment of patient-specific cardiomyocyte sheet using induced pluripotent stem cells generated from adult cardiac fibroblast

scientific article

Disease-specific hematopoietic cell transplantation: nonmyeloablative conditioning regimen for dyskeratosis congenita

scientific article published on 12 April 2010

Dyskeratosis congenita: the first NIH clinical research workshop

scientific article published on September 2009

Early diagnosis of cerebral X-linked adrenoleukodystrophy in boys with Addison's disease improves survival and neurological outcomes

scientific article published on 29 January 2011

Effect of stem cell source on outcomes after unrelated donor transplantation in severe aplastic anemia

scientific article

Elevated cerebral spinal fluid cytokine levels in boys with cerebral adrenoleukodystrophy correlates with MRI severity

scientific article

Engraftment and survival following hematopoietic stem cell transplantation for osteopetrosis using a reduced intensity conditioning regimen

scientific article

Enzyme replacement is associated with better cognitive outcomes after transplant in Hurler syndrome

scientific article

Enzyme replacement therapy prior to haematopoietic stem cell transplantation in Mucopolysaccharidosis Type I: 10 year combined experience of 2 centres

scientific article published on 27 January 2016

Evaluation of TCR Gene Editing Achieved by TALENs, CRISPR/Cas9, and megaTAL Nucleases

scientific article published on 27 October 2015

Expansion and homing of adoptively transferred human natural killer cells in immunodeficient mice varies with product preparation and in vivo cytokine administration: implications for clinical therapy

scientific article

Expression of telomerase and telomere length are unaffected by either age or limb regeneration in Danio rerio

scientific article

Fanconi anemia gene editing by the CRISPR/Cas9 system

scientific article

Fast, Efficient, and Gentle Transfection of Human Adherent Cells in Suspension

scientific article published in April 2016

Favorable outcome of allogeneic hematopoietic cell transplantation for 8p11 myeloproliferative syndrome associated with BCR-FGFR1 gene fusion.

scientific article

Feeder-Free Derivation of Naive Human Pluripotent Stem Cells

scientific article published on 30 June 2017

Fludarabine modulates expression of type VII collagen during haematopoietic stem cell transplantation for recessive dystrophic epidermolysis bullosa

scientific article published on 23 December 2020

Fludarabine-based conditioning for marrow transplantation from unrelated donors in severe aplastic anemia: early results of a cyclophosphamide dose deescalation study show life-threatening adverse events at predefined cyclophosphamide dose levels

scientific article published on 27 April 2012

From Mesoderm to Mesodermatology: Bone Marrow Mesenchymal Cells Heal Skin Wounds

scientific article published on August 2015

From marrow to matrix: novel gene and cell therapies for epidermolysis bullosa

scientific article published on 24 March 2015

GSK3β inhibition activates the CDX/HOX pathway and promotes hemogenic endothelial progenitor differentiation from human pluripotent stem cells.

scientific article published on 16 October 2015

GVHD-associated, inflammasome-mediated loss of function in adoptively transferred myeloid-derived suppressor cells.

scientific article

Gender-related dimorphism in aortic insufficiency in murine mucopolysaccharidosis type I

scientific article

Gene editing and its application for hematological diseases

scientific article published on 27 May 2016

Gene editing toward the use of autologous therapies in recessive dystrophic epidermolysis bullosa

scientific article

Gene therapy for Fanconi anemia: one step closer to the clinic.

scientific article published on February 2012

Genome-wide Specificity of Highly Efficient TALENs and CRISPR/Cas9 for T Cell Receptor Modification

scientific article published on 12 February 2017

Germline mutations in BRCA2: shared genetic susceptibility to breast cancer, early onset leukemia, and Fanconi anemia

scientific article published on 08 January 2004

Granulocyte colony-stimulating factor mobilized CFU-F can be found in the peripheral blood but have limited expansion potential

scientific article published on 9 April 2008

Growth and endocrine function in patients with Hurler syndrome after hematopoietic stem cell transplantation.

scientific article published on 18 February 2008

Hematopoietic Cell Transplantation for Nonmalignant Disorders ⬇️

scientific article published on January 1, 2012

Hematopoietic cell therapy for metabolic disease

scientific article published on October 2007

Hematopoietic cell transplantation for hemoglobinopathies

scientific article published on January 2008

Hematopoietic differentiation of induced pluripotent stem cells from patients with mucopolysaccharidosis type I (Hurler syndrome)

scientific article

Hematopoietic reconstitution by multipotent adult progenitor cells: precursors to long-term hematopoietic stem cells

scientific article

Hematopoietic reconstitution by multipotent adult progenitor cells: precursors to long-term hematopoietic stem cells.

scientific article

Heparin Enhances Transfection in Concert with a Trehalose-Based Polycation with Challenging Cell Types.

scientific article published on 19 December 2016

Higher mycophenolate dose requirements in children undergoing hematopoietic cell transplant (HCT).

scientific article

Hip dysplasia in patients with Hurler syndrome (mucopolysaccharidosis type 1H).

scientific article published on September 2013

Host factors that impact the biodistribution and persistence of multipotent adult progenitor cells

scientific article published on 12 January 2006

Human CD19-Targeted Mouse T Cells Induce B Cell Aplasia and Toxicity in Human CD19 Transgenic Mice

scientific article published on 07 April 2018

IL15 Trispecific Killer Engagers (TriKE) Make Natural Killer Cells Specific to CD33+ Targets While Also Inducing Persistence, In Vivo Expansion, and Enhanced Function

scientific article published on 4 February 2016

Immune regulatory cells in umbilical cord blood: T regulatory cells and mesenchymal stromal cells

scientific article published on October 2009

Immune tolerance of allogeneic hematopoietic cell transplantation supports donor epidermal grafting of recessive dystrophic epidermolysis bullosa chronic wounds

scientific article published on 31 August 2020

Incidence of intracranial germ cell tumors by race in the United States, 1992-2010.

scientific article

Incorporation of Immune Checkpoint Blockade into Chimeric Antigen Receptor T Cells (CAR-Ts): Combination or Built-In CAR-T.

scientific article published on 24 January 2018

Induced Pluripotency and Gene Editing in Fanconi Anemia.

scientific article published on 18 January 2017

Induced pluripotent stem cells from individuals with recessive dystrophic epidermolysis bullosa

scientific article

Induction of comprehensible models for gene expression datasets by subgroup discovery methodology.

scientific article

Inhibiting retinoic acid signaling ameliorates graft-versus-host disease by modifying T-cell differentiation and intestinal migration

scientific article published on 27 June 2013

Inside out: regenerative medicine for recessive dystrophic epidermolysis bullosa.

scientific article published in November 2017

Insights into the mechanism of FTY720 and compatibility with regulatory T cells for the inhibition of graft-versus-host disease (GVHD).

scientific article

Intrathecal enzyme replacement for Hurler syndrome: biomarker association with neurocognitive outcomes

scientific article published on 25 April 2019

Intravenous 6-mercaptopurine decreases salvage after relapse in childhood acute lymphoblastic leukemia: a report from the Children's Cancer Group study CCG 1922.

scientific article published in July 2005

Keratinocytes from induced pluripotent stem cells in junctional epidermolysis bullosa

scientific article

Lactic acidosis and hypoglycemia with ALL relapse following engrafted bone marrow transplant.

scientific article published in August 2009

Large Intragenic Deletion in DSTYK Underlies Autosomal-Recessive Complicated Spastic Paraparesis, SPG23.

scientific article

Late Effects Screening Guidelines after Hematopoietic Cell Transplantation (HCT) for Inherited Bone Marrow Failure Syndromes (IBMFS): Consensus Statement From the Second Pediatric Blood and Marrow Transplant Consortium International Conference on La

scientific article

Leukotriene-receptor inhibition for the treatment of systemic mastocytosis.

scientific article published in February 2004

Lipid Composition of Whole Brain and Cerebellum in Hurler Syndrome (MPS IH) Mice

article

Long-Term Cognitive and Functional Outcomes in Children with Mucopolysaccharidosis (MPS)-IH (Hurler Syndrome) Treated with Hematopoietic Cell Transplantation.

scientific article published on 30 January 2016

Long-term metabolic, endocrine, and neuropsychological outcome of hematopoietic cell transplantation for Wolman disease

scientific article

Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study.

scientific article published on 26 January 2015

Lower leukemia relapse in pediatric patients with pulmonary cytolytic thrombi following allogeneic transplant

scientific article published on 14 June 2010

Lymphoproliferative disorder presenting as pulmonary nodules after bone marrow transplantation

scientific article published on 01 October 2001

Lysyl Hydroxylase 3 Localizes to Epidermal Basement Membrane and Is Reduced in Patients with Recessive Dystrophic Epidermolysis Bullosa

scientific article

MSC therapy attenuates obliterative bronchiolitis after murine bone marrow transplant

scientific article

Management of severe epidermolysis bullosa by haematopoietic transplant: principles, perspectives and pitfalls

scientific article published on 28 September 2012

Marked increased risk of Epstein-Barr virus-related complications with the addition of antithymocyte globulin to a nonmyeloablative conditioning prior to unrelated umbilical cord blood transplantation

scientific article

Mesenchymal stromal cells for graft-versus-host disease

scientific article published on 02 February 2011

Mesenchymal stromal cells from donors varying widely in age are of equal cellular fitness after in vitro expansion under hypoxic conditions

scientific article

Mesenchymal stromal cells inhibit murine syngeneic anti-tumor immune responses by attenuating inflammation and reorganizing the tumor microenvironment

scientific article

Minicircle DNA-based gene therapy coupled with immune modulation permits long-term expression of α-L-iduronidase in mice with mucopolysaccharidosis type I

scientific article

Modeling and rescue of defective blood-brain barrier function of induced brain microvascular endothelial cells from childhood cerebral adrenoleukodystrophy patients.

scientific article

Molecular Additives Significantly Enhance Glycopolymer-Mediated Transfection of Large Plasmids and Functional CRISPR-Cas9 Transcription Activation Ex Vivo in Primary Human Fibroblasts and Induced Pluripotent Stem Cells

scientific article published on 10 December 2018

Montagna Symposium 2017-Precision Dermatology: Next Generation Prevention, Diagnosis, and Treatment.

scientific article published on 14 March 2018

Multipotent adult progenitor cells can suppress graft-versus-host disease via prostaglandin E2 synthesis and only if localized to sites of allopriming

scientific article published on 20 May 2009

Murine chronic graft-versus-host disease proteome profiling discovers CCL15 as a novel biomarker in patients

scientific article published on 18 January 2018

N-acetyl-L-cysteine improves outcome of advanced cerebral adrenoleukodystrophy

scientific article published in February 2007

Non-myeloablative conditioning for second hematopoietic cell transplantation for graft failure in patients with non-malignant disorders: a prospective study and review of the literature

scientific article published on 16 January 2017

On Medawar's 'Actively acquired tolerance of foreign cells'.

scientific article published in February 2014

Optimization of conditioning for marrow transplantation from unrelated donors for patients with aplastic anemia after failure of immunosuppressive therapy

scientific article published on 9 May 2006

Optimization of therapy for severe aplastic anemia based on clinical, biologic, and treatment response parameters: conclusions of an international working group on severe aplastic anemia convened by the Blood and Marrow Transplant Clinical Trials Ne

scientific article

Osteopetrosis

scientific article

Osteopetrosis with micro-lacunar resorption because of defective integrin organization

scientific article

Outcome of haematopoietic stem cell transplantation in dyskeratosis congenita

scientific article published on 09 July 2018

Outcome of penetrating keratoplasty for mucopolysaccharidoses

scientific article published on February 2011

Outcomes after allogeneic hematopoietic cell transplantation for childhood cerebral adrenoleukodystrophy: the largest single-institution cohort report

scientific article published on 17 May 2011

Outcomes of allogeneic hematopoietic cell transplantation in patients with dyskeratosis congenita

scientific article published on 08 June 2013

Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning

scientific article

PAX7 Targets, CD54, Integrin α9β1, and SDC2, Allow Isolation of Human ESC/iPSC-Derived Myogenic Progenitors

scientific article published on June 2017

Patient-specific naturally gene-reverted induced pluripotent stem cells in recessive dystrophic epidermolysis bullosa

scientific article published on 06 December 2013

Pericardial effusion after pediatric hematopoietic cell transplant

article

Peripheral neuro-immune pathology in recessive dystrophic epidermolysis bullosa

scientific article

Pharmacokinetics and Model-Based Dosing to Optimize Fludarabine Therapy in Pediatric Hematopoietic Cell Transplant Recipients

scientific article

Pharmacokinetics of clofarabine in patients with high-risk inherited metabolic disorders undergoing brain-sparing hematopoietic cell transplantation

scientific article

Pharmacologic properties of high-dose ambroxol in four patients with Gaucher disease and myoclonic epilepsy

scientific article published on 24 October 2019

Phase I/II Trial of StemRegenin-1 Expanded Umbilical Cord Blood Hematopoietic Stem Cells Supports Testing as a Stand-Alone Graft

scientific article published on 05 December 2015

Phenotype categorization of neurofibromatosis type I and correlation to NF1 mutation types

scientific article published on 28 November 2019

Placenta-based therapies for the treatment of epidermolysis bullosa

scientific article published on 18 March 2015

Population pharmacokinetics of unbound mycophenolic acid in pediatric and young adult patients undergoing allogeneic hematopoietic cell transplantation

scientific article

Post-transplant laronidase augmentation for children with Hurler syndrome: biochemical outcomes

scientific article published on 01 October 2019

Potential of Systemic Allogeneic Mesenchymal Stromal Cell Therapy for Children with Recessive Dystrophic Epidermolysis Bullosa

scientific article published on 23 April 2015

Pre-transplant risk factors affecting outcome in Hurler syndrome

scientific article published on 9 November 2009

Preclinical Testing of Antihuman CD28 Fab' Antibody in a Novel Nonhuman Primate Small Animal Rodent Model of Xenogenic Graft-Versus-Host Disease

scientific article published in December 2016

Preconditioning of mesenchymal stem cells for improved transplantation efficacy in recessive dystrophic epidermolysis bullosa

scientific article published on 6 November 2014

Prediction of DNA-binding proteins from relational features

scientific article published on 12 November 2012

Program death-1 signaling and regulatory T cells collaborate to resist the function of adoptively transferred cytotoxic T lymphocytes in advanced acute myeloid leukemia.

scientific article

Rapid DNA replication origin licensing protects stem cell pluripotency.

scientific article published on 17 November 2017

Rapid Induction of Cerebral Organoids From Human Induced Pluripotent Stem Cells Using a Chemically Defined Hydrogel and Defined Cell Culture Medium

scientific article published on 13 May 2016

Rapid generation of Col7a1-/- mouse model of recessive dystrophic epidermolysis bullosa and partial rescue via immunosuppressive dermal mesenchymal stem cells

scientific article published on 11 September 2017

Real-time in vivo imaging of stem cells following transgenesis by transposition

scientific article published in July 2005

Regenerative Solutions for Inherited Diseases.

scientific article published on 30 January 2018

Reliability and Validity of iscorEB (Instrument for Scoring Clinical Outcomes of Research for Epidermolysis Bullosa).

scientific article published on 17 January 2018

Reply to: Reduced intensity conditioning haematopoietic stem cell transplantation with mesenchymal stromal cells infusion for the treatment of metachromatic leukodystrophy: a case report. Haematologica 2008; 93:e11-13

scientific article published on 01 May 2008

Response of refractory immune thrombocytopenia after bone marrow transplantation to romiplostim

scientific article published on 01 March 2010

Revertant mosaic fibroblasts in recessive dystrophic epidermolysis bullosa

scientific article published on 08 July 2019

SEGS: search for enriched gene sets in microarray data

scientific article

Safety and early efficacy outcomes for lentiviral fibroblast gene therapy in recessive dystrophic epidermolysis bullosa

scientific article published on 06 June 2019

Sarcoma derived from cultured mesenchymal stem cells

scientific article published on 12 October 2006

Second allogeneic hematopoietic cell transplantation for graft failure: poor outcomes for neutropenic graft failure

scientific article

Semidominant GPNMB Mutations in Amyloidosis Cutis Dyschromica

scientific article published on 19 June 2019

Short-term growth hormone treatment in children with Hurler syndrome after hematopoietic cell transplantation

scientific article

Sickle cell and silent spleen

scientific article published in April 2015

Skeletal muscle contractile function and neuromuscular performance in Zmpste24 -/- mice, a murine model of human progeria

scientific article published on 29 June 2011

Sleeping Beauty transposon-mediated engineering of human primary T cells for therapy of CD19+ lymphoid malignancies

scientific article

Sleeping Beauty-Mediated Transposition in Stem Cells

scholarly article

Sleeping Beauty-mediated correction of Fanconi anemia type C.

scientific article

Stable gene transfer and expression in human primary T cells by the Sleeping Beauty transposon system

scientific article published on 27 September 2005

Stem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting.

scientific article

Stromal cell-derived factor-1 and hematopoietic cell homing in an adult zebrafish model of hematopoietic cell transplantation

scientific article published on 26 May 2011

Synthetic zinc finger nuclease design and rapid assembly

scientific article published on 10 August 2011

T cell progenitor therapy-facilitated thymopoiesis depends upon thymic input and continued thymic microenvironment interaction

scientific article

TALEN-based gene correction for epidermolysis bullosa.

scientific article published on 2 April 2013

TCIRG1 Transgenic Rescue of Osteoclast Function Using Induced Pluripotent Stem Cells Derived from Patients with Infantile Malignant Autosomal Recessive Osteopetrosis

scientific article published on 01 November 2019

TP53 Modulates Oxidative Stress in Gata1+ Erythroid Cells.

scientific article published on 25 January 2017

Targeting of the CNS in MPS-IH using a nonviral transferrin-alpha-L-iduronidase fusion gene product

scientific article published on 03 June 2008

The E5 gene product of rhesus papillomavirus is an activator of endogenous Ras and phosphatidylinositol-3'-kinase in NIH 3T3 cells

scientific article published on November 1996

The host immune response is essential for the beneficial effect of adult stem cells after myocardial ischemia

scientific article published in April 2007

The individual (single patient) IND for inborn errors of metabolism

scientific article published on 13 October 2014

The latent human herpesvirus-6A genome specifically integrates in telomeres of human chromosomes in vivo and in vitro

scientific article

The three-body problem of therapy with induced pluripotent stem cells.

scientific article published on 20 February 2015

Thymic Epithelial Cell Support of Thymopoiesis Does Not Require

scientific article published on 29 October 2018

Toxic Epidermal Necrolysis in Recessive Dystrophic Epidermolysis Bullosa following Bone Marrow Transplantation

scientific article published on 11 March 2016

Translating genome engineering to better clinical outcomes ⬇️

scientific article published on February 15, 2013

Transplacental and Other Routes of Cancer Transmission Between Individuals

scientific article

Transplacental transfer of small-cell carcinoma of the lung

scientific article published on 01 May 2002

Transplant outcomes in leukodystrophies

scientific article published on January 2010

Variability of the deep femoral venous system

scientific article published on 01 January 2011

Venturing into the New Science of Nucleases

scientific article published on April 2016

Zebrafish stromal cells have endothelial properties and support hematopoietic cells

scientific article published on 12 September 2011

alpha-L-iduronidase therapy for mucopolysaccharidosis type I.

scientific article

miR-29 Regulates Type VII Collagen in Recessive Dystrophic Epidermolysis Bullosa.

scientific article published on 17 June 2016

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