Gene therapy for Wiskott-Aldrich syndrome: History, new vectors, future directions

J Allergy Clin Immunol. 2020 Aug;146(2):262-265. doi: 10.1016/j.jaci.2020.06.018. Epub 2020 Jul 2.

¹ San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) and Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.
² Department of Physiology and Biophysics and Institute for Immunology, University of California, Irvine, Calif.
³ San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) and Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy; Vita-Salute San Raffaele University, Milan, Italy.
⁴ Division of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics and Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Stanford, Calif. Electronic address: mg1@stanford.edu.

No abstract available

Keywords: Gene therapy; Wiskott-Aldrich syndrome; hematopoietic stem/progenitor cell; lentiviral vector; reduced-intensity conditioning.

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