Gene therapy for Wiskott-Aldrich syndrome: History, new vectors, future directions - 05/08/20
Doi : 10.1016/j.jaci.2020.06.018
Francesca Ferrua, MD, PhD a, ∗, Francesco Marangoni, PhD b, ∗, Alessandro Aiuti, MD, PhD a, c, ‡, Maria Grazia Roncarolo, MD d, ⁎, ‡ 
a San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) and Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy
b Department of Physiology and Biophysics and Institute for Immunology, University of California, Irvine, Calif
c Vita-Salute San Raffaele University, Milan, Italy
d 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
∗Corresponding author: Maria Grazia Roncarolo, MD, Lorry I. Lokey Stem Cell Research Bldg, 265 Campus Dr West, Rm G3021A, Stanford, CA 94305.Lorry I. Lokey Stem Cell Research Bldg265 Campus Dr WestRm G3021AStanfordCA94305
Key words : Gene therapy, Wiskott-Aldrich syndrome, lentiviral vector, hematopoietic stem/progenitor cell, reduced-intensity conditioning
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| Disclosure of potential conflict of interest: F. Marangoni is supported by a grant from the National Institutes of Health, National Institute of Arthritis and Musculoskeletal and Skin Diseases (grant no. R21 AR072849). The San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) is a joint venture between the Italian Telethon Foundation and Ospedale San Raffaele. Wiskott-Aldrich syndrome gene therapy was licensed to GlaxoSmithKline in 2014 and then transferred to Orchard Therapeutics in April 2018. F. Ferrua and A. Aiuti are investigators of trials nos. NCT01515462 and NCT03837483. |
© 2020
American Academy of Allergy, Asthma & Immunology. Pubblicato da Elsevier Masson SAS. Tutti i diritti riservati.
Vol 146 - N° 2
P. 262-265 - Agosto 2020 Ritorno al numero
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