Utilizing AAV-mediated LEAPER 2.0 for programmable RNA editing in non-human primates and nonsense mutation correction in humanized Hurler syndrome mice.

Zongyi YiYanxia ZhaoZexuan YiYongjian ZhangGangbin TangXiaoxue ZhangHuixian TangWei ZhangYing ZhaoHuayuan XuYuyang NieXueqing SunLijun XingLian DaiPengfei YuanWensheng Wei

Published in: Genome biology (2023)

These discoveries considerably bolster the prospects of employing AAV-borne circular arRNAs for therapeutic applications and exploratory translational research.

Keyphrases

gene therapy
endothelial cells
crispr cas
induced pluripotent stem cells
high fat diet induced
pluripotent stem cells
case report
current status
type diabetes
metabolic syndrome
insulin resistance
skeletal muscle
wild type