Ubiquitous high-level gene expression in hematopoietic lineages provides effective lentiviral gene therapy of murine Wiskott-Aldrich syndrome

Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study

(2011) D. Moratto et al.   BLOOD

Toll-like receptor 7 (TLR7)-driven accumulation of a novel CD11c+ B-cell population is important for the development of autoimmunity

(2011) A. V. Rubtsov et al.   BLOOD

Lentiviral-mediated gene therapy leads to improvement of B-cell functionality in a murine model of Wiskott-Aldrich syndrome

(2011) Marita Bosticardo et al.   JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

Gene therapy for primary immunodeficiencies: Looking ahead, toward gene correction

(2011) Itai M. Pessach et al.   JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

WASp-deficient B cells play a critical, cell-intrinsic role in triggering autoimmunity

(2011) Shirly Becker-Herman et al.   JOURNAL OF EXPERIMENTAL MEDICINE

Solving the Problem of γ-Retroviral Vectors Containing Long Terminal Repeats

(2011) Derek A Persons et al.   MOLECULAR THERAPY

Systemic autoimmunity and defective Fas ligand secretion in the absence of the Wiskott-Aldrich syndrome protein

(2010) N. P. Nikolov et al.   BLOOD

X-linked thrombocytopenia (XLT) due to WAS mutations: clinical characteristics, long-term outcome, and treatment options

(2010) M. H. Albert et al.   BLOOD

B cell-specific lentiviral gene therapy leads to sustained B-cell functional recovery in a murine model of X-linked agammaglobulinemia

(2010) H. M. Kerns et al.   BLOOD

Large-Scale Manufacture and Characterization of a Lentiviral Vector Produced for Clinical Ex Vivo Gene Therapy Application

(2010) Otto-Wilhelm Merten et al.   HUMAN GENE THERAPY

Development of B-lineage Predominant Lentiviral Vectors for Use in Genetic Therapies for B Cell Disorders

(2010) Blythe D Sather et al.   MOLECULAR THERAPY

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

(2010) Marina Cavazzana-Calvo et al.   NATURE

Variants near DMRT1, TERT and ATF7IP are associated with testicular germ cell cancer

(2010) Clare Turnbull et al.   NATURE GENETICS

Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency

(2010) Salima Hacein-Bey-Abina et al.   NEW ENGLAND JOURNAL OF MEDICINE

Stem-Cell Gene Therapy for the Wiskott–Aldrich Syndrome

(2010) Kaan Boztug et al.   NEW ENGLAND JOURNAL OF MEDICINE

Recent advances in understanding the pathophysiology of Wiskott-Aldrich syndrome

(2009) M. Bosticardo et al.   BLOOD

Wiskott-Aldrich syndrome

(2009) Luigi D Notarangelo et al.   CURRENT OPINION IN HEMATOLOGY

Revertant somatic mosaicism in the Wiskott–Aldrich syndrome

(2009) Brian R. Davis et al.   IMMUNOLOGIC RESEARCH

The Wiskott-Aldrich syndrome protein is required for iNKT cell maturation and function

(2009) Michela Locci et al.   JOURNAL OF EXPERIMENTAL MEDICINE

Wiskott-Aldrich Syndrome Protein Is Required for Homeostasis and Function of Invariant NKT Cells

(2009) A. Astrakhan et al.   JOURNAL OF IMMUNOLOGY

Evidence for Long-term Efficacy and Safety of Gene Therapy for Wiskott–Aldrich Syndrome in Preclinical Models

(2009) Francesco Marangoni et al.   MOLECULAR THERAPY

Insertional Transformation of Hematopoietic Cells by Self-inactivating Lentiviral and Gammaretroviral Vectors

(2009) Ute Modlich et al.   MOLECULAR THERAPY

Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency

(2009) Alessandro Aiuti et al.   NEW ENGLAND JOURNAL OF MEDICINE

Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

(2009) N. Cartier et al.   SCIENCE

WASP confers selective advantage for specific hematopoietic cell populations and serves a unique role in marginal zone B-cell homeostasis and function

(2008) L. S. Westerberg et al.   BLOOD

Wiskott-Aldrich syndrome protein deficiency in B cells results in impaired peripheral homeostasis

(2008) A. Meyer-Bahlburg et al.   BLOOD

Improved lentiviral vectors for Wiskott–Aldrich syndrome gene therapy mimic endogenous expression profiles throughout haematopoiesis

(2008) C Frecha et al.   GENE THERAPY

Hematopoietic-Specific Lentiviral Vectors Circumvent Cellular Toxicity Due to Ectopic Expression of Wiskott-Aldrich Syndrome Protein

(2008) Miguel G. Toscano et al.   HUMAN GENE THERAPY

Improvement of Migratory Defects in a Murine Model of Wiskott–Aldrich Syndrome Gene Therapy

(2008) Michael P Blundell et al.   MOLECULAR THERAPY