通过 CRISPR-Cas9 两亲性纳米复合物在体神经元基因编辑可缓解阿尔茨海默病小鼠模型的缺陷。
In vivo neuronal gene editing via CRISPR-Cas9 amphiphilic nanocomplexes alleviates deficits in mouse models of Alzheimer's disease.
机构信息
Department of Biomedical Engineering (BK21 plus), Dongguk University, Seoul, Republic of Korea.
Department of Bioengineering, College of Engineering, Hanyang University, Seoul, Republic of Korea.
出版信息
Nat Neurosci. 2019 Apr;22(4):524-528. doi: 10.1038/s41593-019-0352-0. Epub 2019 Mar 11.
In vivo gene editing in post-mitotic neurons of the adult brain may be a useful strategy for treating neurological diseases. Here, we develop CRISPR-Cas9 nanocomplexes and show they were effective in the adult mouse brain, with minimal off-target effects. Using this system to target Bace1 suppressed amyloid beta (Aβ)-associated pathologies and cognitive deficits in two mouse models of Alzheimer's disease. These results broaden the potential application of CRISPR-Cas9 systems to neurodegenerative diseases.
在成年大脑的有丝分裂后神经元中进行体内基因编辑可能是治疗神经疾病的一种有用策略。在这里，我们开发了 CRISPR-Cas9 纳米复合物，并表明它们在成年小鼠大脑中是有效的，具有最小的脱靶效应。使用该系统靶向 Bace1 可抑制两种阿尔茨海默病小鼠模型中与淀粉样β（Aβ）相关的病理和认知缺陷。这些结果拓宽了 CRISPR-Cas9 系统在神经退行性疾病中的潜在应用。
Zotero 插件