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Liver-directed gene therapy corrects neurologic disease in a murine model of mucopolysaccharidosis type I-Hurler

Mucopolysaccharidosis type I-Hurler (MPS I-H) is a neurodegenerative lysosomal storage disorder (LSD) caused by inherited defects of the α-L-iduronidase (IDUA) gene. Current treatments are ineffective for treating central nervous system (CNS) manifestations because lysosomal enzymes do not effective...

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Detalles Bibliográficos
Autores principales:	Jin, Xiu, Su, Jing, Zhao, Qinyu, Li, Ruiting, Xiao, Jianlu, Zhong, Xiaomei, Song, Li, Liu, Yi, She, Kaiqin, Deng, Hongxin, Wei, Yuquan, Yang, Yang
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2022
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9065053/ https://www.ncbi.nlm.nih.gov/pubmed/35573046 http://dx.doi.org/10.1016/j.omtm.2022.04.010

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