Research ArticleGene Therapy
Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1
- Giulia Schiroli1,2,
- Samuele Ferrari1,2,
- Anthony Conway3,
- Aurelien Jacob1,
- Valentina Capo1,
- Luisa Albano1,
- Tiziana Plati1,
- Maria C. Castiello1,
- Francesca Sanvito4,
- Andrew R. Gennery5,
- Chiara Bovolenta6,
- Rahul Palchaudhuri7,8,
- David T. Scadden8,
- Michael C. Holmes3,
- Anna Villa1,9,
- Giovanni Sitia10,
- Angelo Lombardo1,2,
- Pietro Genovese1,*,† and
- Luigi Naldini1,2,*,†
- 1San Raffaele Telethon Institute for Gene Therapy, 20132 Milan, Italy.
- 2Vita-Salute San Raffaele University, 20132 Milan, Italy.
- 3Sangamo Therapeutics, Richmond, CA 94804, USA.
- 4Pathology Unit, Department of Oncology, San Raffaele Scientific Institute, 20132 Milan, Italy.
- 5Institute of Cellular Medicine, Newcastle University, Newcastle upon Tyne NE2 4HH, UK.
- 6MolMed S.p.A., 20132 Milan, Italy.
- 7Magenta Therapeutics, Cambridge, MA 02139, USA.
- 8Harvard Stem Cell Institute, Cambridge, MA 02138, USA.
- 9National Research Council, Institute of Genetic and Biomedical Research Milan Unit, 20138 Milan, Italy.
- 10San Raffaele Scientific Institute, 20132 Milan, Italy.
- ↵†Corresponding author. Email: genovese.pietro{at}hsr.it (P.G.); naldini.luigi{at}hsr.it (L.N.)
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Science Translational Medicine 11 Oct 2017:
Vol. 9, Issue 411, eaan0820
DOI: 10.1126/scitranslmed.aan0820
