Open AccessNovel AAV-mediated genome editing therapy improves health and survival in a mouse model of methylmalonic acidemia
Author(s) -
Shengwen Zhang,
Amy Bastille,
Susana Gordo,
Nikhil Ramesh,
Jenisha Vora,
Elizabeth McCarthy,
Xiaohan Zhang,
Dylan Frank,
ChihWei Ko,
Carmen Wu,
Noel Walsh,
Shreya Amarwani,
Jing Liao,
Qihua Xiong,
Lauren M. Drouin,
Matthias Hebben,
Kyle P. Chiang,
B Nelson Chau
Publication year - 2022
Publication title -
plos one
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.99
H-Index - 332
ISSN - 1932-6203
DOI - 10.1371/journal.pone.0274774
Subject(s) - methylmalonic acidemia , genome editing , transgene , humanized mouse , biology , genetic enhancement , mutase , microbiology and biotechnology , gene , endocrinology , genome , biochemistry , genetics , in vivo