Improvements of nuclease and nickase gene modification techniques for the treatment of genetic diseases.

Adenosine Base Editor (ABE) CRISPR-cas Cytidine Base Editor (CBE) TALEN ZFN gene editing prime editing

Journal

Frontiers in genome editing
ISSN: 2673-3439
Titre abrégé: Front Genome Ed
Pays: Switzerland
ID NLM: 101775540

Informations de publication

Date de publication:
2022
Historique:
received: 09 03 2022
accepted: 08 07 2022
entrez: 12 8 2022
pubmed: 13 8 2022
medline: 13 8 2022
Statut: epublish

Résumé

Advancements in genome editing make possible to exploit the functions of enzymes for efficient DNA modifications with tremendous potential to treat human genetic diseases. Several nuclease genome editing strategies including Meganucleases (MNs), Zinc Finger Nucleases (ZFNs), Transcription Activator-like Effector Nucleases (TALENs) and Clustered Regularly Interspaced Short Palindromic Repeats-CRISPR associated proteins (CRISPR-Cas) have been developed for the correction of genetic mutations. CRISPR-Cas has further been engineered to create nickase genome editing tools including Base editors and Prime editors with much precision and efficacy. In this review, we summarized recent improvements in nuclease and nickase genome editing approaches for the treatment of genetic diseases. We also highlighted some limitations for the translation of these approaches into clinical applications.

Identifiants

pubmed: 35958050
doi: 10.3389/fgeed.2022.892769
pii: 892769
pmc: PMC9360573
doi:

Types de publication

Journal Article Review

Langues

eng

Pagination

892769

Informations de copyright

Copyright © 2022 Lu, Happi Mbakam, Song, Bendavid and Tremblay.

Déclaration de conflit d'intérêts

The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.

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Auteurs

Yaoyao Lu (Y)

CHU de Québec Research Center, Laval University, Quebec City, QC, Canada.
Department of Molecular Medicine, Laval University, Quebec City, QC, Canada.

Cedric Happi Mbakam (C)

CHU de Québec Research Center, Laval University, Quebec City, QC, Canada.
Department of Molecular Medicine, Laval University, Quebec City, QC, Canada.

Bo Song (B)

CHU de Québec Research Center, Laval University, Quebec City, QC, Canada.
Department of Molecular Medicine, Laval University, Quebec City, QC, Canada.

Eli Bendavid (E)

CHU de Québec Research Center, Laval University, Quebec City, QC, Canada.
Department of Molecular Medicine, Laval University, Quebec City, QC, Canada.

Jacques-P Tremblay (JP)

CHU de Québec Research Center, Laval University, Quebec City, QC, Canada.
Department of Molecular Medicine, Laval University, Quebec City, QC, Canada.

Classifications MeSH