Novel Intravitreal Retina-Targeting and Immune-Evading Adeno-Associated Virus Capsid Variant

How to cite: Cai, Z.; Liu, Y.; Zhao, L.; Li, X.; Zhang, B.; Tang, Y.; Zhou, T.; Zheng, Z.; Li, A.; Wei, J.; Ji, Q.; Lu, D.; Marsic, D. Novel Intravitreal Retina-Targeting and Immune-Evading Adeno-Associated Virus Capsid Variant. Preprints 2024, 2024092179. https://doi.org/10.20944/preprints202409.2179.v1 Cai, Z.; Liu, Y.; Zhao, L.; Li, X.; Zhang, B.; Tang, Y.; Zhou, T.; Zheng, Z.; Li, A.; Wei, J.; Ji, Q.; Lu, D.; Marsic, D. Novel Intravitreal Retina-Targeting and Immune-Evading Adeno-Associated Virus Capsid Variant. Preprints 2024, 2024092179. https://doi.org/10.20944/preprints202409.2179.v1

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MDPI and ACS Style

Cai, Z.; Liu, Y.; Zhao, L.; Li, X.; Zhang, B.; Tang, Y.; Zhou, T.; Zheng, Z.; Li, A.; Wei, J.; Ji, Q.; Lu, D.; Marsic, D. Novel Intravitreal Retina-Targeting and Immune-Evading Adeno-Associated Virus Capsid Variant. Preprints 2024, 2024092179. https://doi.org/10.20944/preprints202409.2179.v1

APA Style

Cai, Z., Liu, Y., Zhao, L., Li, X., Zhang, B., Tang, Y., Zhou, T., Zheng, Z., Li, A., Wei, J., Ji, Q., Lu, D., & Marsic, D. (2024). Novel Intravitreal Retina-Targeting and Immune-Evading Adeno-Associated Virus Capsid Variant. Preprints. https://doi.org/10.20944/preprints202409.2179.v1

Chicago/Turabian Style

Cai, Z., Daru Lu and Damien Marsic. 2024 "Novel Intravitreal Retina-Targeting and Immune-Evading Adeno-Associated Virus Capsid Variant" Preprints. https://doi.org/10.20944/preprints202409.2179.v1

Abstract

Retinal diseases are an important focus of in vivo gene therapy, for which adeno-associated virus is a preferred vector. Intravitreal injection is a highly desirable delivery route because it is much simpler, safer and inexpensive than the main alternative subretinal injection. However, existing capsids have low efficiency, require high doses and are susceptible to neutralization by antibodies that are present in the vitreous. Using AAV5 as starting material, we applied a combination of directed evolution and rational design to develop PT1, a novel AAV capsid capable of efficiently transducing the retina after intravitreal injection. The most striking property of PT1 is its remarkable ability to evade preexisting neutralizing antibodies, thanks to its multiple mutations spread over large areas of the capsid surface, disrupting several epitopes. Our results suggest that PT1 has a strong potential as a useful candidate capsid for human retinal gene therapy.

Keywords

AAV; capsid; intravitreal; retina; immune evasion

Subject

Biology and Life Sciences, Biology and Biotechnology

Copyright: This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

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