Nuclease-Deficient Clustered Regularly Interspaced Short Palindromic Repeat-Based Approaches for In Vitro and In Vivo Gene Activation.

Autor:	Lek A; Department of Genetics, Yale School of Medicine, New Haven, Connecticut, USA., Ma K; Department of Genetics, Yale School of Medicine, New Haven, Connecticut, USA., Woodman KG; Department of Genetics, Yale School of Medicine, New Haven, Connecticut, USA., Lek M; Department of Genetics, Yale School of Medicine, New Haven, Connecticut, USA.
Jazyk:	angličtina
Zdroj:	Human gene therapy [Hum Gene Ther] 2021 Mar; Vol. 32 (5-6), pp. 260-274.
DOI:	10.1089/hum.2020.241
Abstrakt:	Clustered regularly interspaced short palindromic repeat (CRISPR)-based technology has been adapted to achieve a wide range of genome modifications, including transcription regulation. The focus of this review is on the application of CRISPR-based platforms such as nuclease-deficient Cas9 and Cas12a, to achieve targeted gene activation. We review studies to date that have used CRISPR-based activation technology for the elucidation of biological mechanism and disease correction, as well as its application in genetic screens as a powerful tool for high-throughput genotype-phenotype mapping. In addition to our synthesis and critical analysis of published studies, we explore key considerations for the potential clinical translation of CRISPR-based activation technology.
Databáze:	MEDLINE
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