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

Correction of ADA-SCID by Stem Cell Gene Therapy Combined with Nonmyeloablative Conditioning

Alessandro Aiuti, Shimon Slavin, Memet Aker et al. · Science · 2002 · 1.1K citations

Cell Therapy, Immunology, Nonmyeloablative Conditioning +17

Isolation of a novel gene mutated in Wiskott-Aldrich syndrome

Jonathan M.J. Derry, Hans D. Ochs, Uta Francke · Cell · 1994 · 1K citations

Genetic Disorder, Genetics, Pathogenesis +5

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

Salima Hacein‐Bey‐Abina, Frédérique Carlier, Cécile Bouneaud et al. · New England Journal of Medicine · 2002 · 1K citations · Full text

Autoinhibition and activation mechanisms of the Wiskott–Aldrich syndrome protein

Annette S. Kim, Lazaros T. Kakalis, Norzehan Abdul-Manan et al. · Nature · 2000 · 744 citations

Protein Function, Signal Transduction, Disease Mechanism +7

Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration

Eugenio Montini, Daniela Cesana, Manfred Schmidt et al. · Nature Biotechnology · 2006 · 705 citations

Cell Therapy, Medicine, Stem Cell Toxicology +8