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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| Title: | 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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| 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 |
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