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Haematopoietic stem cell transplantation for SCID patients: where do we stand?

British journal of haematology, 2013-01, Vol.160 (2), p.146-152

2013

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Gene therapy for primary adaptive immune deficiencies

Journal of allergy and clinical immunology, 2011-06, Vol.127 (6), p.1356-1359

2011

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Severe combined immunodeficiency. A model disease for molecular immunology and therapy

Immunological reviews, 2005-02, Vol.203 (1), p.98-109

2005

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Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency

The New England journal of medicine, 2010-07, Vol.363 (4), p.355-364

2010

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Gene therapy for primary immunodeficiencies: part 1

Current opinion in immunology, 2012-10, Vol.24 (5), p.580-584

2012

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Gene therapy for primary immunodeficiencies: Part 2

Current opinion in immunology, 2012-10, Vol.24 (5), p.585-591

2012

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Gene therapy of inherited diseases

The Lancet (British edition), 2008-06, Vol.371 (9629), p.2044-2047

2008

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Human T-Lymphoid Progenitors Generated in a Feeder-Cell-Free Delta-Like-4 Culture System Promote T-Cell Reconstitution in NOD/SCID/γc−/− Mice

Stem cells (Dayton, Ohio), 2012-08, Vol.30 (8), p.1771-1780

2012

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Bone Marrow Transplantation for Primary Immunodeficiency Diseases

The Pediatric clinics of North America, 2010-02, Vol.57 (1), p.207-237

2010

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Dynamics of gene-modified progenitor cells analyzed by tracking retroviral integration sites in a human SCID-X1 gene therapy trial

Blood, 2010-06, Vol.115 (22), p.4356-4366

2010

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Characteristics and outcome of early-onset, severe forms of Wiskott-Aldrich syndrome

Blood, 2013-02, Vol.121 (9), p.1510-1516

2013

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Sustained Correction of X-Linked Severe Combined Immunodeficiency by ex Vivo Gene Therapy

The New England journal of medicine, 2002-04, Vol.346 (16), p.1185-1193

2002

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Artemis, a Novel DNA Double-Strand Break Repair/V(D)J Recombination Protein, Is Mutated in Human Severe Combined Immune Deficiency

Cell, 2001-04, Vol.105 (2), p.177-186

2001

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Gene Therapy of Human Severe Combined Immunodeficiency (SCID)-X1 Disease

Science (American Association for the Advancement of Science), 2000-04, Vol.288 (5466), p.669-672

2000

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A method to sequence and quantify DNA integration for monitoring outcome in gene therapy

Nucleic acids research, 2011-06, Vol.39 (11), p.e72

2011

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Primary Microcephaly, Impaired DNA Replication, and Genomic Instability Caused by Compound Heterozygous ATR Mutations

Human mutation, 2013-02, Vol.34 (2), p.374-384

2013

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Hematopoietic stem cell transplantation in hemophagocytic lymphohistiocytosis: a single-center report of 48 patients

Pediatrics (Evanston), 2006-04, Vol.117 (4), p.e743

2006

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Severe combined immunodeficiency and microcephaly in siblings with hypomorphic mutations in DNA ligase IV

European Journal of Immunology, 2006-01, Vol.36 (1), p.224-235

2006

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Is normal hematopoiesis maintained solely by long-term multipotent stem cells?

Blood, 2011-04, Vol.117 (17), p.4420-4424

2011

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Bone Marrow Transplantation Attenuates the Myopathic Phenotype of a Muscular Mouse Model of Spinal Muscular Atrophy

Stem cells (Dayton, Ohio), 2006-12, Vol.24 (12), p.2723-2732

2006

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Autoimmunity in Wiskott-Aldrich syndrome: risk factors, clinical features, and outcome in a single-center cohort of 55 patients

Pediatrics (Evanston), 2003-05, Vol.111 (5 Pt 1), p.e622

2003

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Allogeneic hematopoietic stem-cell transplantation for leukocyte adhesion deficiency

Pediatrics (Evanston), 2009-03, Vol.123 (3), p.836

2009

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Gene therapy for severe combined immunodeficiency

Annual review of medicine, 2005-02, Vol.56 (1), p.585-602

2005

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Haematopoietic stem cell transplantation for SCID patients: where do we stand?

Gene therapy for primary adaptive immune deficiencies

Severe combined immunodeficiency. A model disease for molecular immunology and therapy

Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency

Gene therapy for primary immunodeficiencies: part 1

Gene therapy for primary immunodeficiencies: Part 2

Gene therapy of inherited diseases

Human T-Lymphoid Progenitors Generated in a Feeder-Cell-Free Delta-Like-4 Culture System Promote T-Cell Reconstitution in NOD/SCID/γc−/− Mice

Bone Marrow Transplantation for Primary Immunodeficiency Diseases

Dynamics of gene-modified progenitor cells analyzed by tracking retroviral integration sites in a human SCID-X1 gene therapy trial

Characteristics and outcome of early-onset, severe forms of Wiskott-Aldrich syndrome

Sustained Correction of X-Linked Severe Combined Immunodeficiency by ex Vivo Gene Therapy

Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy

Artemis, a Novel DNA Double-Strand Break Repair/V(D)J Recombination Protein, Is Mutated in Human Severe Combined Immune Deficiency

Gene Therapy of Human Severe Combined Immunodeficiency (SCID)-X1 Disease

A method to sequence and quantify DNA integration for monitoring outcome in gene therapy

Primary Microcephaly, Impaired DNA Replication, and Genomic Instability Caused by Compound Heterozygous ATR Mutations

Hematopoietic stem cell transplantation in hemophagocytic lymphohistiocytosis: a single-center report of 48 patients

Long-term outcome following hematopoietic stem-cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and European Group for Blood and Marrow Transplantation

Severe combined immunodeficiency and microcephaly in siblings with hypomorphic mutations in DNA ligase IV

Is normal hematopoiesis maintained solely by long-term multipotent stem cells?

Bone Marrow Transplantation Attenuates the Myopathic Phenotype of a Muscular Mouse Model of Spinal Muscular Atrophy

Autoimmunity in Wiskott-Aldrich syndrome: risk factors, clinical features, and outcome in a single-center cohort of 55 patients

Allogeneic hematopoietic stem-cell transplantation for leukocyte adhesion deficiency

Gene therapy for severe combined immunodeficiency

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