Key result
Small Cas protein orthologs suitable for single AAV vector delivery expand translational applications and genetic sites that can be targeted for in vivo gene editing of inherited diseases.
Why the study?
AAV vectors for CRISPR/Cas gene editing are limited by packaging capacity, making identification of smaller Cas orthologs suitable for single AAV delivery necessary.
Utilizing small Cas orthologs for single AAV delivery overcomes packaging limitations, expanding the potential for in vivo CRISPR/Cas gene editing therapies.
Answers are grounded in this paper and cite their sources.
May facilitate AAV-CRISPR editing for inherited cardiovascular diseases; leaves open clinical validation and safety data.
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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Stevanovic et al. (2021) conducted a review in Inherited diseases. Small Cas protein orthologs for single AAV vector delivery was evaluated. Small Cas protein orthologs suitable for single AAV vector delivery expand translational applications and genetic sites that can be targeted for in vivo gene editing of inherited diseases.
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