Customizable virus-like particles deliver CRISPR-Cas9 ribonucleoprotein for effective ocular neovascular and Huntington's disease gene therapy.

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Tác giả: Pinar Akcakaya, Bilada Bilican, Yujia Cai, Yao Dai, Mike Firth, Jiaxu Hong, Zhuofan Jiang, Sikai Ling, Jianping Liu, Sicong Lu, Anders Lundin, Xiaoqing Qian, Tugce Munise Satir, Niklas Selfjord, Julia Liz Touza, Natalie Van Zuydam, Xue Zhang, Xujiao Zhou

Ngôn ngữ: eng

Ký hiệu phân loại: 518.6 Numerical methods in analysis

Thông tin xuất bản: England : Nature nanotechnology , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 51127

In vivo CRISPR gene editing holds enormous potential for various diseases. Ideally, CRISPR delivery should be cell type-specific and time-restricted for optimal efficacy and safety, but customizable methods are lacking. Here we develop a cell-tropism programmable CRISPR-Cas9 ribonucleoprotein delivery system (RIDE) based on virus-like particles. The efficiency of RIDE was comparable to that of adeno-associated virus and lentiviral vectors and higher than lipid nanoparticles. RIDE could be readily reprogrammed to target dendritic cells, T cells and neurons, and significantly ameliorated the disease symptoms in both ocular neovascular and Huntington's disease models via cell-specific gene editing. In addition, RIDE could efficiently edit the huntingtin gene in patients' induced pluripotent stem cell-derived neurons and was tolerated in non-human primates. This study is expected to facilitate the development of in vivo CRISPR therapeutics.
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