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- W4308792354 endingPage "500" @default.
- W4308792354 startingPage "471" @default.
- W4308792354 abstract "Advancements in genome editing enable permanent changes of DNA sequences in a site-specific manner, providing promising approaches for treating human genetic disorders caused by gene mutations. Recently, genome editing has been applied and achieved significant progress in treating inherited genetic disorders that remain incurable by conventional therapy. Here, we present a review of various programmable genome editing systems with their principles, advantages, and limitations. We introduce their recent applications for treating inherited diseases in the clinic, including sickle cell disease (SCD), β-thalassemia, Leber congenital amaurosis (LCA), heterozygous familial hypercholesterolemia (HeFH), etc. We also discuss the paradigm of ex vivo and in vivo editing and highlight the promise of somatic editing and the challenge of germline editing. Finally, we propose future directions in delivery, cutting, and repairing to improve the scope of clinical applications." @default.
- W4308792354 created "2022-11-15" @default.
- W4308792354 creator A5004498551 @default.
- W4308792354 creator A5053780153 @default.
- W4308792354 creator A5083153699 @default.
- W4308792354 date "2022-10-01" @default.
- W4308792354 modified "2023-10-15" @default.
- W4308792354 title "Site-specific genome editing in treatment of inherited diseases: possibility, progress, and perspectives" @default.
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