In vivo prime editing rescues photoreceptor degeneration in nonsense mutant retinitis pigmentosa.

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Tác giả: Peiwei Chai, Xianqun Fan, Yidian Fu, Xin D Gao, Shengfang Ge, Xiaoyu He, Renbing Jia, David R Liu, Pengpeng Liu, Liang Ma, Hanhan Shi, Zhi Yang

Ngôn ngữ: eng

Ký hiệu phân loại: 121.6 Nature of inquiry

Thông tin xuất bản: England : Nature communications , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 686092

The next-generation gene editing tool, prime editing (PE), is adept at correcting point mutations precisely with high editing efficiency and rare off-target events and shows promising therapeutic value in treating hereditary diseases. Retinitis pigmentosa (RP) is the most common type of inherited retinal dystrophy and is characterized by progressive degeneration of retinal photoreceptors and, consequently, visual decline. To date, effective treatments for RP are lacking. Herein, a PE system is designed to target the PDE6B Y347X mutation in the rd1 mouse strain, a preclinical RP model. We screen and develop the PE system with epegRNA and RT
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