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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Autors principals:	Xiu Jin, Jing Su, Qinyu Zhao, Ruiting Li, Jianlu Xiao, Xiaomei Zhong, Li Song, Yi Liu, Kaiqin She, Hongxin Deng, Yuquan Wei, Yang Yang
Format:	Artigo
Idioma:	Inglês
Publicat:	Elsevier 2022-06-01
Col·lecció:	Molecular Therapy: Methods & Clinical Development
Matèries:	MPS I Hurler syndrome CNS disease MTfp AAV gene therapy
Accés en línia:	http://www.sciencedirect.com/science/article/pii/S2329050122000584
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