Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1-Reference-Cited by-同舟云学术

Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1

Published:2017-10-11 Issue:411 Volume:9 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Schiroli Giulia¹²^ORCID,Ferrari Samuele¹²^ORCID,Conway Anthony³,Jacob Aurelien¹^ORCID,Capo Valentina¹^ORCID,Albano Luisa¹,Plati Tiziana¹^ORCID,Castiello Maria C.¹,Sanvito Francesca⁴,Gennery Andrew R.⁵,Bovolenta Chiara⁶,Palchaudhuri Rahul⁷⁸^ORCID,Scadden David T.⁸^ORCID,Holmes Michael C.³,Villa Anna¹⁹^ORCID,Sitia Giovanni¹⁰^ORCID,Lombardo Angelo¹²^ORCID,Genovese Pietro¹^ORCID,Naldini Luigi¹²^ORCID

Affiliation:

1. San Raffaele Telethon Institute for Gene Therapy, 20132 Milan, Italy.

2. Vita-Salute San Raffaele University, 20132 Milan, Italy.

3. Sangamo Therapeutics, Richmond, CA 94804, USA.

4. Pathology Unit, Department of Oncology, San Raffaele Scientific Institute, 20132 Milan, Italy.

5. Institute of Cellular Medicine, Newcastle University, Newcastle upon Tyne NE2 4HH, UK.

6. MolMed S.p.A., 20132 Milan, Italy.

7. Magenta Therapeutics, Cambridge, MA 02139, USA.

8. Harvard Stem Cell Institute, Cambridge, MA 02138, USA.

9. National Research Council, Institute of Genetic and Biomedical Research Milan Unit, 20138 Milan, Italy.

10. San Raffaele Scientific Institute, 20132 Milan, Italy.

Abstract

Preclinical studies establish the conditions for safe and effective correction of SCID-X1 by targeted gene editing of hematopoietic stem cells.

Funder

Seventh Framework Programme

Fondazione Telethon

Ministero della Salute

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

Reference43 articles.

1. The new frontier of genome engineering with CRISPR-Cas9

2. A guide to genome engineering with programmable nucleases

3. Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery

4. In vivo genome editing restores haemostasis in a mouse model of haemophilia

5. Promoterless gene targeting without nucleases ameliorates haemophilia B in mice

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