In vivo genome editing improves motor function and extends survival in a mouse model of ALS-Reference-Cited by-同舟云学术

In vivo genome editing improves motor function and extends survival in a mouse model of ALS

Author:

Gaj Thomas¹^ORCID,Ojala David S.²,Ekman Freja K.³^ORCID,Byrne Leah C.⁴^ORCID,Limsirichai Prajit⁵,Schaffer David V.¹²⁴^ORCID

Affiliation:

1. Department of Bioengineering, University of California, Berkeley, Berkeley, CA 94720, USA.

2. Department of Chemical and Biomolecular Engineering, University of California, Berkeley, Berkeley, CA 94720, USA.

3. Department of Chemistry, University of California, Berkeley, Berkeley, CA 94720, USA.

4. Helen Wills Neuroscience Institute, University of California, Berkeley, Berkeley, CA 94720, USA.

5. Department of Plant and Microbial Biology, University of California, Berkeley, Berkeley, CA 94720, USA.

Abstract

CRISPR-Cas9–mediated genome editing can be used to treat ALS in an animal model of the disease.

Funder

National Institutes of Health

NSF Graduate Research Fellowship

UC Berkeley Dissertation-Year Fellowship

Publisher

American Association for the Advancement of Science (AAAS)

Subject

Multidisciplinary

Reference84 articles.

1. Amyotrophic lateral sclerosis;Rowland L. P.;N. Engl. J. Med.,2001

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