激活剂(遗传学)
Cas9
清脆的
体内
生物
细胞生物学
基因
遗传学
作者
Hanseul Park,Yerim Hwang,Jong-Pil Kim
出处
期刊:Biomaterials
[Elsevier]
日期:2021-12-01
卷期号:279: 121229-121229
被引量:7
标识
DOI:10.1016/j.biomaterials.2021.121229
摘要
CRISPR/Cas9-mediated gene activation is a potential therapeutic strategy that does not induce double-strand break (DSB) DNA damage. However, in vivo gene activation via a Cas9 activator remains a challenge, currently limiting its therapeutic applications. We developed a Cas9 activator nanocomplex that efficiently activates an endogenous gene in the brain in vivo, suggesting its possible application in novel therapeutics. We demonstrated a potential treatment application of the Cas9 activator nanocomplex by activating Adam10 in the mouse brain without introducing insertions and deletions (inDels). Remarkably, in vivo activation of Adam10 with the Cas9 activator nanocomplex improved cognitive deficits in an Alzheimer's disease (AD) mouse model. These results demonstrate the therapeutic potential of Cas9 activator nanocomplexes for a wide range of neurological diseases.
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