Ten years of gene therapy for primary immune deficiencies

Recent advances in understanding the pathophysiology of Wiskott-Aldrich syndrome

(2009) M. Bosticardo et al.   BLOOD

Long-term outcome after hematopoietic stem cell transplantation of a single-center cohort of 90 patients with severe combined immunodeficiency

(2009) B. Neven et al.   BLOOD

Revertant somatic mosaicism in the Wiskott–Aldrich syndrome

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

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

A Novel Model of SCID-X1 Reconstitution Reveals Predisposition to Retrovirus-induced Lymphoma but No Evidence of γC Gene Oncogenicity

(2009) Linda Scobie et al.   MOLECULAR THERAPY

Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency

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

Murine Leukemias with Retroviral Insertions at Lmo2 Are Predictive of the Leukemias Induced in SCID-X1 Patients Following Retroviral Gene Therapy

(2009) Utpal P. Davé et al.   PLoS Genetics

Neonatal bone marrow transplantation of ADA-deficient SCID mice results in immunologic reconstitution despite low levels of engraftment and an absence of selective donor T lymphoid expansion

(2008) D. A. Carbonaro et al.   BLOOD

Altered intracellular and extracellular signaling leads to impaired T-cell functions in ADA-SCID patients

(2008) B. Cassani et al.   BLOOD

Modern management of chronic granulomatous disease

(2008) Reinhard A. Seger   BRITISH JOURNAL OF HAEMATOLOGY

Development of lentiviral gene therapy for Wiskott Aldrich syndrome

(2008) Anne Galy et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Advances in treatment for chronic granulomatous disease

(2008) Elizabeth M. Kang et al.   IMMUNOLOGIC RESEARCH

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

Gene therapy of inherited diseases

(2008) Alain Fischer et al.   LANCET

Self-inactivating Gammaretroviral Vectors for Gene Therapy of X-linked Severe Combined Immunodeficiency

(2008) Susannah I Thornhill et al.   MOLECULAR THERAPY