Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome

Lentiviral gene therapy corrects platelet phenotype and function in Wiskott-Aldrich patients

(2019) Lucia Sereni et al.   JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

Author Correction: Gene correction for SCID-X1 in long-term hematopoietic stem cells

(2019) Mara Pavel-Dinu et al.   Nature Communications

Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study

(2019) Francesca Ferrua et al.   Lancet Haematology

Comparison of Zinc Finger Nucleases Versus CRISPR-Specific Nucleases for Genome Editing of the Wiskott-Aldrich Syndrome Locus

(2018) Alejandra Gutierrez-Guerrero et al.   HUMAN GENE THERAPY

Autonomous role of Wiskott-Aldrich syndrome platelet deficiency in inducing autoimmunity and inflammation

(2018) Lucia Sereni et al.   JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

Site-Specific Gene Editing of Human Hematopoietic Stem Cells for X-Linked Hyper-IgM Syndrome

(2018) Caroline Y. Kuo et al.   Cell Reports

A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells

(2018) Christopher A. Vakulskas et al.   NATURE MEDICINE

CRISPR-Cas9 gene repair of hematopoietic stem cells from patients with X-linked chronic granulomatous disease

(2017) Suk See De Ravin et al.   Science Translational Medicine

Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1

(2017) Giulia Schiroli et al.   Science Translational Medicine

Megakaryocyte Differentiation and Platelet Formation from Human Cord Blood-derived CD34+ Cells

(2017) Jose Perdomo et al.   Jove-Journal of Visualized Experiments

Linkage between the mechanisms of thrombocytopenia and thrombopoiesis

(2016) K. Eto et al.   BLOOD

Open-source guideseq software for analysis of GUIDE-seq data

(2016) Shengdar Q Tsai et al.   NATURE BIOTECHNOLOGY

Analyzing CRISPR genome-editing experiments with CRISPResso

(2016) Luca Pinello et al.   NATURE BIOTECHNOLOGY

Gene Correction of iPSCs from a Wiskott-Aldrich Syndrome Patient Normalizes the Lymphoid Developmental and Functional Defects

(2016) Tamara J. Laskowski et al.   Stem Cell Reports

Current and emerging treatment options for Wiskott–Aldrich syndrome

(2015) Austen JJ Worth et al.   Expert Review of Clinical Immunology

Outcomes Following Gene Therapy in Patients With Severe Wiskott-Aldrich Syndrome

(2015) Salima Hacein-Bey Abina et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells

(2015) Ayal Hendel et al.   NATURE BIOTECHNOLOGY

Targeted genome editing in human repopulating haematopoietic stem cells

(2014) Pietro Genovese et al.   NATURE

GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases

(2014) Shengdar Q Tsai et al.   NATURE BIOTECHNOLOGY

Gene Therapy for Wiskott-Aldrich Syndrome--Long-Term Efficacy and Genotoxicity

(2014) C. J. Braun et al.   Science Translational Medicine

COSMID: A Web-based Tool for Identifying and Validating CRISPR/Cas Off-target Sites

(2014) Thomas J Cradick et al.   Molecular Therapy-Nucleic Acids

Mechanisms of Retroviral Integration and Mutagenesis

(2013) Alessia Cavazza et al.   HUMAN GENE THERAPY

Wiskott–Aldrich Syndrome protein deficiency perturbs the homeostasis of B-cell compartment in humans

(2013) Maria Carmina Castiello et al.   JOURNAL OF AUTOIMMUNITY

Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome

(2013) A. Aiuti et al.   SCIENCE

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

(2012) A. Astrakhan et al.   BLOOD

Outcomes following hematopoietic cell transplantation for Wiskott–Aldrich syndrome

(2012) C R Shin et al.   BONE MARROW TRANSPLANTATION

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

Isolation of Single Human Hematopoietic Stem Cells Capable of Long-Term Multilineage Engraftment

(2011) F. Notta et al.   SCIENCE

Stem-Cell Gene Therapy for the Wiskott–Aldrich Syndrome

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

Wiskott-Aldrich Syndrome: Diagnosis, Clinical and Laboratory Manifestations, and Treatment

(2009) Hans D. Ochs et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Wiskott-Aldrich syndrome

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

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