Correction of RNA splicing defect in beta(654)-thalassemia mice using CRISPR/Cas9 gene-editing technology

Lu, D; Gong, XL; Fang, YD; Guo, XB; Chen, YW; Yang, F; Zhao, GJ; Ma, QW; Zeng, YT; Zeng, FY

Zeng, FY (通讯作者)，Shanghai Jiao Tong Univ, Shanghai Childrens Hosp, Shanghai Inst Med Genet, Shanghai, Peoples R China.;Zeng, FY (通讯作者)，Shanghai Jiao Tong Univ, Dept Histoembryol Genet & Dev Biol, Sch Med, Shanghai, Peoples R China.;Zeng, FY (通讯作者)，Minist Hlth, Key Lab Embryo Mol Biol, Shanghai, Peoples R China.;Zeng, FY (通讯作者)，Shanghai Key Lab Embryo & Reprod Engn, Shanghai, Peoples R China.

HAEMATOLOGICA, 2022; 107 (6): 1427

Abstract

beta(654)-thalassemia is a prominent Chinese subtype of beta-thalassemia, representing 17% of all cases of beta-thalassemia in China. The molecular me......

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