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Whole-body galactose oxidation as a robust functional assay to assess the efficacy of gene-based therapies in a mouse model of Galactosemia.
Mol Ther Methods Clin Dev. 2024 Jan 17;32(1):101191. doi: 10.1016/j.omtm.2024.101191. eCollection 2024 Mar 14.
Mol Ther Methods Clin Dev. 2024.
PMID: 38352271
Free PMC article.
AAV-based gene therapy prevents and halts the progression of dilated cardiomyopathy in a mouse model of phosphoglucomutase 1 deficiency (PGM1-CDG).
Balakrishnan B, Altassan R, Budhraja R, Liou W, Lupo A, Bryant S, Mankouski A, Radenkovic S, Preston GJ, Pandey A, Boudina S, Kozicz T, Morava E, Lai K.
Balakrishnan B, et al.
Transl Res. 2023 Jul;257:1-14. doi: 10.1016/j.trsl.2023.01.004. Epub 2023 Jan 26.
Transl Res. 2023.
PMID: 36709920
Free PMC article.
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