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Molecular Evidence of Genome Editing in a Mouse Model of Immunodeficiency

Scientific reports, 2018-05, Vol.8 (1), p.8214-13, Article 8214

2018

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DNA delivery to ‘ex vivo’ human liver segments

Gene therapy, 2012-05, Vol.19 (5), p.504-512

2012

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Generation of iPSCs from Genetically Corrected Brca2 Hypomorphic Cells: Implications in Cell Reprogramming and Stem Cell Therapy

Stem cells (Dayton, Ohio), 2014-02, Vol.32 (2), p.436-446

2014

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CD34+AC133+ Cells Isolated from Cord Blood are Highly Enriched in Long‐Term Culture‐Initiating Cells, NOD/SCID‐Repopulating Cells and Dendritic Cell Progenitors

Stem cells (Dayton, Ohio), 1998-11, Vol.16 (6), p.387-396

1998

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GSE4 peptide suppresses oxidative and telomere deficiencies in ataxia telangiectasia patient cells

Cell death and differentiation, 2019-10, Vol.26 (10), p.1998-2014

2019

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Efficient Non-viral Gene Delivery into Human Hematopoietic Stem Cells by Minicircle Sleeping Beauty Transposon Vectors

Molecular therapy, 2018-04, Vol.26 (4), p.1137-1153

2018

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Engraftment and in vivo proliferation advantage of gene-corrected mobilized CD34+ cells from Fanconi anemia patients

Blood, 2017-09, Vol.130 (13), p.1535-1542

2017

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Distinct classes of human stem cells that differ in proliferative and self-renewal potential

Nature immunology, 2001-01, Vol.2 (1), p.75-82

2001

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Lentiviral-Mediated Gene Therapy in Fanconi Anemia-A Mice Reveals Long-Term Engraftment and Continuous Turnover of Corrected HSCs

Current gene therapy, 2015-01, Vol.15 (6), p.550-562

2015

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Delayed Engraftment of Nonobese Diabetic/Severe Combined Immunodeficient Mice Transplanted With Ex Vivo–Expanded Human CD34+ Cord Blood Cells

Blood, 1999-02, Vol.93 (3), p.1097-1105

1999

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Development of lentiviral vectors with optimized transcriptional activity for the gene therapy of patients with Fanconi anemia

Human gene therapy, 2010-05, Vol.21 (5), p.623-630

2010

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Delayed Engraftment of Nonobese Diabetic/Severe Combined Immunodeficient Mice Transplanted With Ex Vivo–Expanded Human CD34+ Cord Blood Cells

Blood, 1999-02, Vol.93 (3), p.1097-1105

1999

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Transduction of Human CD34+CD38- Bone Marrow and Cord Blood-Derived SCID-Repopulating Cells with Third-Generation Lentiviral Vectors

Molecular therapy, 2000-06, Vol.1 (6), p.566-573

2000

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AM218, a new polyanionic polysaccharide, induces radioprotection in mice when administered shortly before irradiation

International journal of radiation biology, 1997, Vol.71 (1), p.101-108

GUENECHEA, G.

1997

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Parvovirus Infection Suppresses Long-Term Repopulating Hematopoietic Stem Cells

Journal of Virology, 2003-08, Vol.77 (15), p.8495-8503

2003

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Regulatory elements of the vav gene drive transgene expression in hematopoietic stem cells from adult mice

Experimental hematology, 2004-04, Vol.32 (4), p.360-364

2004

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Ex vivo expansion of umbilical cord blood (UCB) CD34(+) cells alters the expression and function of alpha 4 beta 1 and alpha 5 beta 1 integrins

British journal of haematology, 2001-10, Vol.115 (1), p.213-221

2001

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Preclinical gene therapy studies of fanconi anemia with lentiviral vectors

Blood cells, molecules, & diseases, 2008-03, Vol.40 (2), p.258-258

2008

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In Vivo Dynamics of Human Stem Cell Repopulation in NOD/SCID Mice

Annals of the New York Academy of Sciences, 2001-06, Vol.938 (1), p.184-190

2001

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Significant impact of mesenchymal stromal cell co-transplantation in a challenging hematopoietic engraftment scenario comparing two administration routes

Cytotherapy (Oxford, England), 2020-05, Vol.22 (5), p.S60-S60

2020

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Efficient transduction of human hematopoietic repopulating cells generating stable engraftment of transgene-expressing cells in NOD/SCID mice

Blood, 2000-05, Vol.95 (10), p.3085-3093

2000

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Preserved long-term repopulation and differentiation properties of hematopoietic grafts subjected to ex vivo expansion with stem cell factor and interleukin 11

Transplantation, 1999-05, Vol.67 (10), p.1348

1999

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Generation and potential applications of an X-linked dyskeratosis congenita model in human hematopoietic stem cells

IBJ Plus, 2018-05

2018

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Genetic modification of hematopoietic stem cells: recent advances in the gene therapy of inherited diseases

Archives of Medical Research, 2003-11, Vol.34 (6), p.589-599

2003

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Systematic analysis of clinically applicable conditions leading to a high efficiency of transduction and transgene expression in human T cells

The journal of gene medicine, 2001-01, Vol.3 (1), p.32-41

2001

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Molecular Evidence of Genome Editing in a Mouse Model of Immunodeficiency

DNA delivery to ‘ex vivo’ human liver segments

Generation of iPSCs from Genetically Corrected Brca2 Hypomorphic Cells: Implications in Cell Reprogramming and Stem Cell Therapy

CD34+AC133+ Cells Isolated from Cord Blood are Highly Enriched in Long‐Term Culture‐Initiating Cells, NOD/SCID‐Repopulating Cells and Dendritic Cell Progenitors

GSE4 peptide suppresses oxidative and telomere deficiencies in ataxia telangiectasia patient cells

Efficient Non-viral Gene Delivery into Human Hematopoietic Stem Cells by Minicircle Sleeping Beauty Transposon Vectors

Engraftment and in vivo proliferation advantage of gene-corrected mobilized CD34+ cells from Fanconi anemia patients

Distinct classes of human stem cells that differ in proliferative and self-renewal potential

Lentiviral-Mediated Gene Therapy in Fanconi Anemia-A Mice Reveals Long-Term Engraftment and Continuous Turnover of Corrected HSCs

Delayed Engraftment of Nonobese Diabetic/Severe Combined Immunodeficient Mice Transplanted With Ex Vivo–Expanded Human CD34+ Cord Blood Cells

Development of lentiviral vectors with optimized transcriptional activity for the gene therapy of patients with Fanconi anemia

Delayed Engraftment of Nonobese Diabetic/Severe Combined Immunodeficient Mice Transplanted With Ex Vivo–Expanded Human CD34+ Cord Blood Cells

Transduction of Human CD34+CD38- Bone Marrow and Cord Blood-Derived SCID-Repopulating Cells with Third-Generation Lentiviral Vectors

AM218, a new polyanionic polysaccharide, induces radioprotection in mice when administered shortly before irradiation

Parvovirus Infection Suppresses Long-Term Repopulating Hematopoietic Stem Cells

Regulatory elements of the vav gene drive transgene expression in hematopoietic stem cells from adult mice

Ex vivo expansion of umbilical cord blood (UCB) CD34(+) cells alters the expression and function of alpha 4 beta 1 and alpha 5 beta 1 integrins

Preclinical gene therapy studies of fanconi anemia with lentiviral vectors

In Vivo Dynamics of Human Stem Cell Repopulation in NOD/SCID Mice

Significant impact of mesenchymal stromal cell co-transplantation in a challenging hematopoietic engraftment scenario comparing two administration routes

Efficient transduction of human hematopoietic repopulating cells generating stable engraftment of transgene-expressing cells in NOD/SCID mice

Preserved long-term repopulation and differentiation properties of hematopoietic grafts subjected to ex vivo expansion with stem cell factor and interleukin 11

Generation and potential applications of an X-linked dyskeratosis congenita model in human hematopoietic stem cells

Genetic modification of hematopoietic stem cells: recent advances in the gene therapy of inherited diseases

Systematic analysis of clinically applicable conditions leading to a high efficiency of transduction and transgene expression in human T cells

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