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AAV-based CRISPR-Cas9 genome editing: Challenges and engineering opportunities

Publication ,  Journal Article
Kabadi, AM; Mejia-Guerra, MK; Graef, JD; Khan, SZ; Walton, EM; Wang, X; Gersbach, CA; Potter, R
Published in: Current Opinion in Biomedical Engineering
March 1, 2024

Recent innovations in the field of gene therapy have paved the way for advances towards developing genome editing medicines. Despite these steps forward, challenges with viral delivery of genome editing tools persist. Efforts currently underway include developing next-generation genome editors, overcoming adeno-associated virus (AAV) packaging restrictions, improving AAV genome integrity, engineering novel AAV capsids, and minimizing the immune response. This review discusses current challenges in delivering CRISPR-Cas nuclease-based genome editing therapies using AAV and highlights emerging methods to overcome these obstacles. This includes developing smaller payloads and regulatory elements, advancing novel sequencing methods for vector characterization, engineering capsids with enhanced potency, tissue-selectivity, and ability to evade pre-existing antibodies, controlling transgene expression, and minimizing the immune response to Cas proteins.

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Published In

Current Opinion in Biomedical Engineering

DOI

EISSN

2468-4511

Publication Date

March 1, 2024

Volume

29

Related Subject Headings

  • 4003 Biomedical engineering
 

Citation

APA
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ICMJE
MLA
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Kabadi, A. M., Mejia-Guerra, M. K., Graef, J. D., Khan, S. Z., Walton, E. M., Wang, X., … Potter, R. (2024). AAV-based CRISPR-Cas9 genome editing: Challenges and engineering opportunities. Current Opinion in Biomedical Engineering, 29. https://doi.org/10.1016/j.cobme.2023.100517
Kabadi, A. M., M. K. Mejia-Guerra, J. D. Graef, S. Z. Khan, E. M. Walton, X. Wang, C. A. Gersbach, and R. Potter. “AAV-based CRISPR-Cas9 genome editing: Challenges and engineering opportunities.” Current Opinion in Biomedical Engineering 29 (March 1, 2024). https://doi.org/10.1016/j.cobme.2023.100517.
Kabadi AM, Mejia-Guerra MK, Graef JD, Khan SZ, Walton EM, Wang X, et al. AAV-based CRISPR-Cas9 genome editing: Challenges and engineering opportunities. Current Opinion in Biomedical Engineering. 2024 Mar 1;29.
Kabadi, A. M., et al. “AAV-based CRISPR-Cas9 genome editing: Challenges and engineering opportunities.” Current Opinion in Biomedical Engineering, vol. 29, Mar. 2024. Scopus, doi:10.1016/j.cobme.2023.100517.
Kabadi AM, Mejia-Guerra MK, Graef JD, Khan SZ, Walton EM, Wang X, Gersbach CA, Potter R. AAV-based CRISPR-Cas9 genome editing: Challenges and engineering opportunities. Current Opinion in Biomedical Engineering. 2024 Mar 1;29.
Journal cover image

Published In

Current Opinion in Biomedical Engineering

DOI

EISSN

2468-4511

Publication Date

March 1, 2024

Volume

29

Related Subject Headings

  • 4003 Biomedical engineering