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113 - Lentiviral-Mediated Gene Therapy for Patients with Fanconi Anemia [Group A]: Updated Results from Global RP-L102 Clinical Trials

Transplantation and cellular therapy, 2023-02, Vol.29 (2), p.S89-S89

2023

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228. Characterization of Hematopoietic Progenitors from Pyruvate Kinase Deficient (PKD) Patients and Transduction of PKD CD34+ Cells with a Therapeutic Lentiviral Vector

Molecular therapy, 2016-05, Vol.24, p.S89-S89

2016

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293 - Lentiviral-Mediated Gene Therapy for Severe Pyruvate Kinase Deficiency: Results from an Ongoing Global Phase 1 Study

Transplantation and cellular therapy, 2023-02, Vol.29 (2), p.S225-S226

2023

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3 - Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results from an Ongoing Phase 1/2 Study

Transplantation and cellular therapy, 2023-02, Vol.29 (2), p.S2-S3

2023

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558. Targeted Gene Therapy in CD34+ Cells from Healthy Donors and Fanconi Anemia Patients

Molecular therapy, 2016-05, Vol.24, p.S223-S223

2016

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753. Lentiviral-Mediated Gene Correction of Mobilized Peripheral Blood Progenitors and Repopulating Cells from FA-A Patients

Molecular therapy, 2016-05, Vol.24, p.S297-S298

2016

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Access to gene therapy for rare diseases when commercialization is not fit for purpose

Nature medicine, 2023-03, Vol.29 (3), p.518-519

2023

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Adenine base editing efficiently restores the function of Fanconi anemia hematopoietic stem and progenitor cells

Nature communications, 2022-11, Vol.13 (1), p.6900-6900, Article 6900

2022

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Adipose‐derived mesenchymal stromal cells modulate experimental autoimmune arthritis by inducing an early regulatory innate cell signature

Immunity, Inflammation and Disease, 2016-06, Vol.4 (2), p.213-224

2016

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Adipose Tissue‐Derived Mesenchymal Stem Cells Have In Vivo Immunosuppressive Properties Applicable for the Control of the Graft‐Versus‐Host Disease

Stem cells (Dayton, Ohio), 2006-11, Vol.24 (11), p.2582-2591

2006

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Advances and Challenges in the Development of Gene Therapy Medicinal Products for Rare Diseases

Human gene therapy, 2023-09, Vol.34 (17-18), p.763-775

2023

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Advances in Gene Therapy for Fanconi Anemia

Human gene therapy, 2018-10, Vol.29 (10), p.1114-1123

2018

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Advances in the gene therapy of monogenic blood cell diseases

Clinical genetics, 2020-01, Vol.97 (1), p.89-102

2020

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Advances in the Gene Therapy of Patients with Fanconi Anemia

Blood, 2018-11, Vol.132 (Supplement 1), p.1022-1022

2018

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α-Glucan Recognition by a New Family of Carbohydrate-Binding Modules Found Primarily in Bacterial Pathogens

Biochemistry (Easton), 2004-12, Vol.43 (49), p.15633-15642

2004

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AM5, a protein-associated polysaccharide, stimulates hematopoiesis and modulates the expression of endogenous hematopoietic growth factors in murine long-term bone marrow cultures

Stem cells (Dayton, Ohio), 1995-03, Vol.13 (2), p.175-185

1995

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Bcr/Abl interferes with the Fanconi anemia/BRCA pathway: implications in the chromosomal instability of chronic myeloid leukemia cells

PloS one, 2010-12, Vol.5 (12), p.e15525-e15525

2010

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BCR-JAK2 drives a myeloproliferative neoplasm in transplanted mice

The Journal of pathology, 2015-06, Vol.236 (2), p.219-228

2015

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Biochemical Correction of X-CGD by a Novel Chimeric Promoter Regulating High Levels of Transgene Expression in Myeloid Cells

Molecular therapy, 2011-01, Vol.19 (1), p.122-132

2011

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Biodistribution and Efficacy of Human Adipose-Derived Mesenchymal Stem Cells Following Intranodal Administration in Experimental Colitis

Frontiers in immunology, 2017-06, Vol.8, p.638-638

2017

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Bone marrow-derived cells promote liver regeneration in mice with erythropoietic protoporphyria

Transplantation, 2009-12, Vol.88 (12), p.1332-1340

2009

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Brief Report: Impaired Cell Reprogramming in Nonhomologous End Joining Deficient Cells

Stem cells (Dayton, Ohio), 2013-08, Vol.31 (8), p.1726-1730

2013

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Brief Report: Reduced Expression of CD18 Leads to the In Vivo Expansion of Hematopoietic Stem Cells in Mouse Bone Marrow

Stem cells (Dayton, Ohio), 2014-10, Vol.32 (10), p.2794-2798

2014

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Cell cycle-dependent chromatin loading of the Fanconi anemia core complex by FANCM/FAAP24. Commentary

Blood, 2008, Vol.111 (10)

2008

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Cell Therapy With Mesenchymal Stem Cells Induces an Innate Immune Memory Response That Attenuates Experimental Colitis in the Long Term

Journal of Crohn's and colitis, 2020-10, Vol.14 (10), p.1424-1435

2020

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113 - Lentiviral-Mediated Gene Therapy for Patients with Fanconi Anemia [Group A]: Updated Results from Global RP-L102 Clinical Trials

228. Characterization of Hematopoietic Progenitors from Pyruvate Kinase Deficient (PKD) Patients and Transduction of PKD CD34+ Cells with a Therapeutic Lentiviral Vector

293 - Lentiviral-Mediated Gene Therapy for Severe Pyruvate Kinase Deficiency: Results from an Ongoing Global Phase 1 Study

3 - Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results from an Ongoing Phase 1/2 Study

558. Targeted Gene Therapy in CD34+ Cells from Healthy Donors and Fanconi Anemia Patients

753. Lentiviral-Mediated Gene Correction of Mobilized Peripheral Blood Progenitors and Repopulating Cells from FA-A Patients

Access to gene therapy for rare diseases when commercialization is not fit for purpose

Adenine base editing efficiently restores the function of Fanconi anemia hematopoietic stem and progenitor cells

Adipose‐derived mesenchymal stromal cells modulate experimental autoimmune arthritis by inducing an early regulatory innate cell signature

Adipose Tissue‐Derived Mesenchymal Stem Cells Have In Vivo Immunosuppressive Properties Applicable for the Control of the Graft‐Versus‐Host Disease

Advances and Challenges in the Development of Gene Therapy Medicinal Products for Rare Diseases

Advances in Gene Therapy for Fanconi Anemia

Advances in the gene therapy of monogenic blood cell diseases

Advances in the Gene Therapy of Patients with Fanconi Anemia

α-Glucan Recognition by a New Family of Carbohydrate-Binding Modules Found Primarily in Bacterial Pathogens

AM5, a protein-associated polysaccharide, stimulates hematopoiesis and modulates the expression of endogenous hematopoietic growth factors in murine long-term bone marrow cultures

Bcr/Abl interferes with the Fanconi anemia/BRCA pathway: implications in the chromosomal instability of chronic myeloid leukemia cells

BCR-JAK2 drives a myeloproliferative neoplasm in transplanted mice

Biochemical Correction of X-CGD by a Novel Chimeric Promoter Regulating High Levels of Transgene Expression in Myeloid Cells

Biodistribution and Efficacy of Human Adipose-Derived Mesenchymal Stem Cells Following Intranodal Administration in Experimental Colitis

Bone marrow-derived cells promote liver regeneration in mice with erythropoietic protoporphyria

Brief Report: Impaired Cell Reprogramming in Nonhomologous End Joining Deficient Cells

Brief Report: Reduced Expression of CD18 Leads to the In Vivo Expansion of Hematopoietic Stem Cells in Mouse Bone Marrow

Cell cycle-dependent chromatin loading of the Fanconi anemia core complex by FANCM/FAAP24. Commentary

Cell Therapy With Mesenchymal Stem Cells Induces an Innate Immune Memory Response That Attenuates Experimental Colitis in the Long Term

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