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Systemic gene therapy corrects the neurological phenotype in a mouse model of NGLY1 deficiency.

Du A, Yang K, Zhou X, Ren L, Liu N, Zhou C, Liang J, Yan N, Gao G, Wang D. Systemic gene therapy corrects the neurological phenotype in a mouse model of NGLY1 deficiency. JCI Insight. 2024 Oct 08; 9(19).

View in: PubMed