Reporter Mice for Gene Editing: A Key Tool for Advancing Gene Therapy of Rare Diseases

Autor:	Siang Li, Cord Brakebusch
Jazyk:	angličtina
Rok vydání:	2024
Předmět:	gene editing reporter mice CRISPR rare diseases Cytology QH573-671
Zdroj:	Cells, Vol 13, Iss 17, p 1508 (2024)
Druh dokumentu:	article
ISSN:	2073-4409
DOI:	10.3390/cells13171508
Popis:	Most rare diseases are caused by mutations and can have devastating consequences. Precise gene editing by CRISPR/Cas is an exciting possibility for helping these patients, if no irreversible developmental defects have occurred. To optimize gene editing therapy, reporter mice for gene editing have been generated which, by expression of reporter genes, indicate the efficiency of precise and imprecise gene editing. These mice are important tools for testing and comparing novel gene editing methodologies. This review provides a comprehensive overview of reporter mice for gene editing which all have been used for monitoring CRISPR/Cas-mediated gene editing involving DNA double-strand breaks (DSBs). Furthermore, we discuss how reporter mice can be used for quickly checking genetic alterations by base editing (BE) or prime editing (PE).
Databáze:	Directory of Open Access Journals
Externí odkaz:	https://doaj.org/article/83b07bc589bc40e1bd0819e940b848bd Zobrazit plný text záznamu View record in DOAJ Plný text ve formátu PDF Plný text ve formátu HTML
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