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...

Descripción completa

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

Internet

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

Ejemplares similares