Enhancing RNA editing efficiency and specificity with engineered ADAR2 guide RNAs.

 0 Người đánh giá. Xếp hạng trung bình 0

Tác giả: Xilei Ai, Xin Cui, Sheng Ding, Juan Dong, Feng Du, Xin Huang, Shuai Liu, Zhuo Tang, Shan Zhou

Ngôn ngữ: eng

Ký hiệu phân loại: 920.71 Men

Thông tin xuất bản: United States : Molecular therapy. Nucleic acids , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 210186

RNA editing is a prospective therapeutic approach for correcting harmful mutations, offering the benefits of reversibility and tunability without permanently modifying the genome. However, the relatively low enzymatic activity and the occurrence of off-target editing events present significant challenges, limiting its utility. In response to this limitation, we introduced a novel strategy: strand displacement-responsive ADAR system for RNA editing (SPRING) by adding a "blocking sequence" to form a hairpin guide RNA. This modification significantly improves the efficiency of site-directed RNA editing (SDRE) at various target sites. Furthermore, the use of hairpin guide RNA within the SPRING system enhances the specificity of RNA editing through competitive reactions during target hybridization. In principle, this approach can be employed across various ADAR-based editing systems, offering a novel RNA-editing platform with wide-ranging potential for research, therapy, and biotech applications.
Tạo bộ sưu tập với mã QR

THƯ VIỆN - TRƯỜNG ĐẠI HỌC CÔNG NGHỆ TP.HCM

ĐT: (028) 36225755 | Email: tt.thuvien@hutech.edu.vn

Copyright @2024 THƯ VIỆN HUTECH