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 10 08; 9(19).