AAV-based gene replacement therapy prevents and halts manifestation of abnormal neurological phenotypes in a novel mouse model of PMM2-CDG.

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Bibliographic Details
Title: AAV-based gene replacement therapy prevents and halts manifestation of abnormal neurological phenotypes in a novel mouse model of PMM2-CDG.
Authors: Zhong ML; Division of Medical Genetics, Department of Pediatrics, University of Utah Spencer Fox Eccles School of Medicine, Salt Lake City, UT, USA., Lai K; Division of Medical Genetics, Department of Pediatrics, University of Utah Spencer Fox Eccles School of Medicine, Salt Lake City, UT, USA. kent.lai@hsc.utah.edu.
Source: Gene therapy [Gene Ther] 2025 May; Vol. 32 (3), pp. 246-254. Date of Electronic Publication: 2025 Mar 17.
Publication Type: Journal Article; Research Support, Non-U.S. Gov't; Research Support, N.I.H., Extramural
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/s41434-025-00525-w