Using RNA-targeting CRISPR-Cas13 and engineered U1 systems to target ABCA4 splice variants in Stargardt disease
利用靶向RNA的CRISPR-Cas13和工程化的U1系统靶向Stargardt病中的ABCA4剪接变体
期刊:Molecular Therapy-Nucleic Acids
影响因子:6.5
doi:10.1016/j.omtn.2025.102789.
Roxanne Hsiang-Chi Liou,Daniel Urrutia-Cabrera,Chia-Fei Liu,Sihui Wu,Ida Maria Westin,Irina Golovleva,Guei-Sheung Liu,Satheesh Kumar,Samuel McLenachan,Fred Kuanfu Chen,Fei-Ting Hsu,Chien-Ling Huang,Tom Edwards,Keith R Martin,Albert Wu Cheng,Raymond C B Wong
