PR001 gene therapy increased GCase activity and improved Gaucher disease type 1 phenotypes in mouse models

Autor:	Zhaohui Yang, Sid Kamalakaran, Asa Abeliovich, Jason Politi, Laura D. Heckman, Swetha Garimalla, Franz Hefti, Hsuan-Ni Lin, Li Chin Wong, Patty Sheehan, Yong Dai
Rok vydání:	2021
Předmět:	Endocrinology Endocrinology Diabetes and Metabolism Genetic enhancement Genetics Cancer research Disease Biology Molecular Biology Biochemistry Phenotype
Zdroj:	Molecular Genetics and Metabolism. 132:S100
ISSN:	1096-7192
DOI:	10.1016/j.ymgme.2020.12.242
Databáze:	OpenAIRE
Externí odkaz:	https://explore.openaire.eu/search/publication?articleId=doi_________::bc634f2fbead049ca80c0b6b4af0187b https://doi.org/10.1016/j.ymgme.2020.12.242 Zobrazit plný text záznamu Full Text from ScienceDirect