Engineering adeno-associated viral vectors for CRISPR/Cas based in vivo therapeutic genome editing

Apr 9, 2025Biomaterials

Designing viral tools to deliver CRISPR/Cas for gene editing treatments inside the body

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Abstract

The recent FDA approval of the first gene editing therapy marks a significant milestone in treating sickle cell disease and transfusion-dependent beta-thalassemia.

CRISPR technologies show potential for addressing previously incurable genetic disorders.
Ex vivo gene editing approaches have advanced significantly, while in vivo therapies face challenges in efficient delivery.
Adeno-associated viral (AAV) vectors are promising for delivering gene editing tools but have limitations.
Engineering strategies are being developed to improve the efficiency, specificity, and safety of AAV-CRISPR systems.

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The recent approval of the first gene editing therapy for sickle cell disease and transfusion-dependent beta-thalassemia by the U.S. Food and Drug Administration (FDA) demonstrates the immense potential of CRISPR (clustered regularly interspaced short palindromic repeats) technologies to treat patients with genetic disorders that were previously considered incurable. While significant advancements have been made with ex vivo gene editing approaches, the development of in vivo CRISPR/Cas gene editing therapies has not progressed as rapidly due to significant challenges in achieving highly efficient and specific in vivo delivery. Adeno-associated viral (AAV) vectors have shown great promise in clinical trials as vehicles for delivering therapeutic transgenes and other cargos but currently face multiple limitations for effective delivery of gene editing machineries. This review elucidates these challenges and highlights the latest engineering strategies aimed at improving the efficiency, specificity, and safety profiles of AAV-packaged CRISPR/Cas systems (AAV-CRISPR) to enhance their clinical utility.

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Engineering adeno-associated viral vectors for CRISPR/Cas based in vivo therapeutic genome editing

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