Journal
CURRENT GENE THERAPY
Volume 22, Issue 1, Pages 1-14Publisher
BENTHAM SCIENCE PUBL LTD
DOI: 10.2174/1566523221666211006120355
Keywords
CRISPR; CRISPR; Cas9; gene therapy; Cas9; orthologs; AAV
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Funding
- Howard Hughes Medical Institute
- Foundation Fighting Blindness - NIHR Oxford Biomedical Research Centre
- Retina UK
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CRISPR/Cas gene editing is a revolutionary technology for correcting genetic mutations in vivo. AAV vectors are potential tools for delivering CRISPR/Cas, but their limited capacity is a restriction. Identifying smaller Cas orthologs that can be packaged into a single AAV would optimize gene editing and expand the targeted genetic sites.
CRISPR (clustered regularly interspaced short palindromic repeats)/Cas gene editing is a revolutionary technology that can enable the correction of genetic mutations in vivo, providing great promise as a therapeutic intervention for inherited diseases. Adeno-associated viral (AAV) vectors are a potential vehicle for delivering CRISPR/Cas. However, they are restricted by their limited packaging capacity. Identifying smaller Cas orthologs that can be packaged, along with the required guide RNA elements, into a single AAV would be an important optimization for CRISPR/Cas gene editing. Expanding the options of Cas proteins that can be delivered by a single AAV not only increases translational application but also expands the genetic sites that can be targeted for editing. This review considers the benefits and current scope of small Cas protein orthologs that are suitable for gene editing approaches using single AAV vector delivery.
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