CNS-targeted base editing of the major late-onset Tay-Sachs mutation alleviates disease in mice

Journal:

Journal of Clinical Investigation

Published:

2025-06-17

DOI:

10.1172/jci183434

Affiliations:

2

Authors:

13

Go to article

Institutions	Authors	Share
NIH National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), United States of America (USA)	12.000000 12.000000	0.92
NIH National Human Genome Research Institute (NHGRI), United States of America (USA)	1.000000	0.08

Interested in Medical Biochemistry and Metabolomics?