A solid start for gene therapy in Tay–Sachs disease

A ‘gene addition’ strategy shows promise in a first-in-human trial involving two infant patients.

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Bibliographic Details
Published in:	Nature medicine Vol. 28; no. 2; pp. 236 - 237
Main Authors:	Yu, Timothy W., Bodamer, Olaf
Format:	Journal Article
Language:	English
Published:	New York Nature Publishing Group US 01-02-2022 Nature Publishing Group
Subjects:	692/308/153 692/308/575 692/617/375/2764 Acute coronary syndromes Amino acids Biomedical and Life Sciences Biomedicine Cancer Research Cardiovascular disease Drug dosages Enzymes Gene therapy Genetic Therapy Heart attacks Humans Infectious Diseases Medicine Metabolic Diseases Metabolism Metabolites Molecular Medicine Mutation Neurological diseases Neurosciences News & Views news-and-views Patients Spinal cord Tay-Sachs disease Tay-Sachs Disease - genetics Tay-Sachs Disease - therapy Vectors (Biology)
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Description
Summary:	A ‘gene addition’ strategy shows promise in a first-in-human trial involving two infant patients.
Bibliography:	ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 ObjectType-Commentary-3 content type line 23
ISSN:	1078-8956 1546-170X
DOI:	10.1038/s41591-022-01687-5