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Research ArticleGene Therapy
Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1
- View ORCID ProfileGiulia Schiroli1,2,
- View ORCID ProfileSamuele Ferrari1,2,
- Anthony Conway3,
- View ORCID ProfileAurelien Jacob1,
- View ORCID ProfileValentina Capo1,
- Luisa Albano1,
- View ORCID ProfileTiziana Plati1,
- Maria C. Castiello1,
- Francesca Sanvito4,
- Andrew R. Gennery5,
- Chiara Bovolenta6,
- View ORCID ProfileRahul Palchaudhuri7,8,
- View ORCID ProfileDavid T. Scadden8,
- Michael C. Holmes3,
- View ORCID ProfileAnna Villa1,9,
- View ORCID ProfileGiovanni Sitia10,
- View ORCID ProfileAngelo Lombardo1,2,
- View ORCID ProfilePietro Genovese1,*,† and
- View ORCID ProfileLuigi 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.)
↵* These authors contributed equally to this work.
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Science Translational Medicine 11 Oct 2017:
Vol. 9, Issue 411, eaan0820
DOI: 10.1126/scitranslmed.aan0820
Vol. 9, Issue 411, eaan0820
DOI: 10.1126/scitranslmed.aan0820
Giulia Schiroli
1San Raffaele Telethon Institute for Gene Therapy, 20132 Milan, Italy.
2Vita-Salute San Raffaele University, 20132 Milan, Italy.
Samuele Ferrari
1San Raffaele Telethon Institute for Gene Therapy, 20132 Milan, Italy.
2Vita-Salute San Raffaele University, 20132 Milan, Italy.
Anthony Conway
3Sangamo Therapeutics, Richmond, CA 94804, USA.
Aurelien Jacob
1San Raffaele Telethon Institute for Gene Therapy, 20132 Milan, Italy.
Valentina Capo
1San Raffaele Telethon Institute for Gene Therapy, 20132 Milan, Italy.
Luisa Albano
1San Raffaele Telethon Institute for Gene Therapy, 20132 Milan, Italy.
Tiziana Plati
1San Raffaele Telethon Institute for Gene Therapy, 20132 Milan, Italy.
Maria C. Castiello
1San Raffaele Telethon Institute for Gene Therapy, 20132 Milan, Italy.
Francesca Sanvito
4Pathology Unit, Department of Oncology, San Raffaele Scientific Institute, 20132 Milan, Italy.
Andrew R. Gennery
5Institute of Cellular Medicine, Newcastle University, Newcastle upon Tyne NE2 4HH, UK.
Chiara Bovolenta
6MolMed S.p.A., 20132 Milan, Italy.
Rahul Palchaudhuri
7Magenta Therapeutics, Cambridge, MA 02139, USA.
8Harvard Stem Cell Institute, Cambridge, MA 02138, USA.
David T. Scadden
8Harvard Stem Cell Institute, Cambridge, MA 02138, USA.
Michael C. Holmes
3Sangamo Therapeutics, Richmond, CA 94804, USA.
Anna Villa
1San Raffaele Telethon Institute for Gene Therapy, 20132 Milan, Italy.
9National Research Council, Institute of Genetic and Biomedical Research Milan Unit, 20138 Milan, Italy.
Giovanni Sitia
10San Raffaele Scientific Institute, 20132 Milan, Italy.
Angelo Lombardo
1San Raffaele Telethon Institute for Gene Therapy, 20132 Milan, Italy.
2Vita-Salute San Raffaele University, 20132 Milan, Italy.
Pietro Genovese
1San Raffaele Telethon Institute for Gene Therapy, 20132 Milan, Italy.
Luigi Naldini
1San Raffaele Telethon Institute for Gene Therapy, 20132 Milan, Italy.
2Vita-Salute San Raffaele University, 20132 Milan, Italy.
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