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

Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

(2013) A. Biffi et al.   SCIENCE

Wiskott-Aldrich syndrome protein (WASP) and N-WASP are critical for peripheral B-cell development and function

(2012) L. S. Westerberg et al.   BLOOD

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

(2012) A. Astrakhan et al.   BLOOD

Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans

(2012) F. Candotti et al.   BLOOD

Outcomes following hematopoietic cell transplantation for Wiskott–Aldrich syndrome

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

Gene therapy for primary immunodeficiencies: part 1

(2012) Marina Cavazzana-Calvo et al.   CURRENT OPINION IN IMMUNOLOGY

Gene therapy for primary immunodeficiencies: Part 2

(2012) Alessandro Aiuti et al.   CURRENT OPINION IN IMMUNOLOGY

Challenges in Vector and Trial Design Using Retroviral Vectors for Long-Term Gene Correction in Hematopoietic Stem Cell Gene Therapy

(2012) Jacqueline Corrigan-Curay et al.   MOLECULAR THERAPY

Retroviral Integrations in Gene Therapy Trials

(2012) Luca Biasco et al.   MOLECULAR THERAPY

Gene therapy matures in the clinic

(2012) Leonard W Seymour et al.   NATURE BIOTECHNOLOGY

Counting stem cells: methodological constraints

(2012) Leonid V Bystrykh et al.   NATURE METHODS

Autoimmunity in Wiskott–Aldrich Syndrome: An Unsolved Enigma

(2012) Marco Catucci et al.   Frontiers in Immunology

Alterations in the adenosine metabolism and CD39/CD73 adenosinergic machinery cause loss of Treg cell function and autoimmunity in ADA-deficient SCID

(2011) A. V. Sauer et al.   BLOOD

Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection

(2011) A. Biffi et al.   BLOOD

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

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

IL-2 induces a WAVE2-dependent pathway for actin reorganization that enables WASp-independent human NK cell function

(2011) Jordan S. Orange et al.   JOURNAL OF CLINICAL INVESTIGATION

Insertion Sites in Engrafted Cells Cluster Within a Limited Repertoire of Genomic Areas After Gammaretroviral Vector Gene Therapy

(2011) Annette Deichmann et al.   MOLECULAR THERAPY

Ex vivo gene transfer and correction for cell-based therapies

(2011) Luigi Naldini   NATURE REVIEWS GENETICS

Analyzing the Number of Common Integration Sites of Viral Vectors – New Methods and Computer Programs

(2011) Ulrich Abel et al.   PLoS One

Long-Term Persistence of a Polyclonal T Cell Repertoire After Gene Therapy for X-Linked Severe Combined Immunodeficiency

(2011) H. B. Gaspar et al.   Science Translational Medicine

High-definition mapping of retroviral integration sites identifies active regulatory elements in human multipotent hematopoietic progenitors

(2010) C. Cattoglio et al.   BLOOD

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

(2010) G. P. Wang et al.   BLOOD

Integration profile of retroviral vector in gene therapy treated patients is cell-specific according to gene expression and chromatin conformation of target cell

(2010) Luca Biasco et al.   EMBO Molecular Medicine

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

(2010) Marina Cavazzana-Calvo et al.   NATURE

GREAT improves functional interpretation of cis-regulatory regions

(2010) Cory Y McLean et al.   NATURE BIOTECHNOLOGY

Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease

(2010) Stefan Stein et al.   NATURE MEDICINE

Genome-wide high-throughput integrome analyses by nrLAM-PCR and next-generation sequencing

(2010) Anna Paruzynski et al.   Nature Protocols

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

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

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

Recent advances in understanding the pathophysiology of Wiskott-Aldrich syndrome

(2009) M. Bosticardo et al.   BLOOD

How I treat ADA deficiency

(2009) H. B. Gaspar et al.   BLOOD

Chromatin Signatures in Multipotent Human Hematopoietic Stem Cells Indicate the Fate of Bivalent Genes during Differentiation

(2009) Kairong Cui et al.   Cell Stem Cell

Wiskott-Aldrich syndrome

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

The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy

(2009) Eugenio Montini et al.   JOURNAL OF CLINICAL INVESTIGATION

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

Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

(2008) Steven J. Howe et al.   JOURNAL OF CLINICAL INVESTIGATION

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

(2008) Salima Hacein-Bey-Abina et al.   JOURNAL OF CLINICAL INVESTIGATION