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Efficacy of an AAV vector encoding a thermostable form of glucocerebrosidase in alleviating symptoms in a Gaucher disease mouse model.

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Title: Efficacy of an AAV vector encoding a thermostable form of glucocerebrosidase in alleviating symptoms in a Gaucher disease mouse model.
Authors: Milenkovic I; Department of Biomolecular Sciences, Weizmann Institute of Science, Rehovot, 7610001, Israel.; Department of Neurology, Medical University of Vienna, Vienna, Austria., Blumenreich S; Department of Biomolecular Sciences, Weizmann Institute of Science, Rehovot, 7610001, Israel., Hochfelder A; Department of Biomolecular Sciences, Weizmann Institute of Science, Rehovot, 7610001, Israel., Azulay A; Department of Biomolecular Sciences, Weizmann Institute of Science, Rehovot, 7610001, Israel., Biton IE; Department of Veterinary Resources, Weizmann Institute of Science, Rehovot, 7610001, Israel., Zerbib M; Department of Veterinary Resources, Weizmann Institute of Science, Rehovot, 7610001, Israel., Oren R; Department of Veterinary Resources, Weizmann Institute of Science, Rehovot, 7610001, Israel., Tsoory M; Department of Veterinary Resources, Weizmann Institute of Science, Rehovot, 7610001, Israel., Joseph T; Department of Biomolecular Sciences, Weizmann Institute of Science, Rehovot, 7610001, Israel., Fleishman SJ; Department of Biomolecular Sciences, Weizmann Institute of Science, Rehovot, 7610001, Israel., Futerman AH; Department of Biomolecular Sciences, Weizmann Institute of Science, Rehovot, 7610001, Israel. tony.futerman@weizmann.ac.il.
Source: Gene therapy [Gene Ther] 2024 Sep; Vol. 31 (9-10), pp. 439-444. Date of Electronic Publication: 2024 Aug 15.
Publication Type: Journal Article; Research Support, Non-U.S. Gov't
Journal Info: Publisher: Nature Publishing Group Country of Publication: England NLM ID: 9421525 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1476-5462 (Electronic) Linking ISSN: 09697128 NLM ISO Abbreviation: Gene Ther Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:1476-5462
DOI:10.1038/s41434-024-00476-8