Site-specific genome editing in treatment of inherited diseases: possibility, progress, and perspectives
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.
- Standort
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Deutsche Nationalbibliothek Frankfurt am Main
- Umfang
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Online-Ressource
- Sprache
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Englisch
- Erschienen in
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Site-specific genome editing in treatment of inherited diseases: possibility, progress, and perspectives ; volume:2 ; number:5 ; year:2022 ; pages:471-500 ; extent:030
Medical Review ; 2, Heft 5 (2022), 471-500 (gesamt 030)
- Urheber
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Huang, Chao
Li, Qing
Li, Jinsong
- DOI
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10.1515/mr-2022-0029
- URN
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urn:nbn:de:101:1-2022120213055824434785
- Rechteinformation
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Open Access; Der Zugriff auf das Objekt ist unbeschränkt möglich.
- Letzte Aktualisierung
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15.08.2025, 07:27 MESZ
Datenpartner
Deutsche Nationalbibliothek. Bei Fragen zum Objekt wenden Sie sich bitte an den Datenpartner.
Beteiligte
- Huang, Chao
- Li, Qing
- Li, Jinsong
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