The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV2-Reference-Cited by-同舟云学术

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The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV2

Published:2024-08-12 Issue:9-10 Volume:31 Page:489-498
ISSN:0969-7128
Container-title:Gene Therapy
language:en
Short-container-title:Gene Ther

Author:

Cui Mengtian,Su Qin,Yip Mitchell,McGowan Jackson,Punzo Claudio,Gao Guangping^ORCID,Tai Phillip W. L.^ORCID

Funder

The Bassick Family Foundation Award, BRIDGE Fund Award

U.S. Department of Health & Human Services | NIH | National Institute of Neurological Disorders and Stroke

U.S. Department of Health & Human Services | NIH | National Institute of Allergy and Infectious Diseases

U.S. Department of Health & Human Services | NIH | National Heart, Lung, and Blood Institute

Publisher

Springer Science and Business Media LLC

Link

https://www.nature.com/articles/s41434-024-00477-7.pdf

Reference63 articles.

1. Bucher K, Rodriguez-Bocanegra E, Dauletbekov D, Fischer MD. Immune responses to retinal gene therapy using adeno-associated viral vectors - Implications for treatment success and safety. Prog Retin Eye Res. 2021;83:100915.

2. Kuzmin DA, Shutova MV, Johnston NR, Smith OP, Fedorin VV, Kukushkin YS, et al. The clinical landscape for AAV gene therapies. Nat Rev Drug Discov. 2021;20:173–4.

3. Wang D, Tai PWL, Gao G. Adeno-associated virus vector as a platform for gene therapy delivery. Nat Rev Drug Discov. 2019;18:358–78.

4. Sarkar H, Moosajee M. Choroideremia: molecular mechanisms and therapies. Trends Mol Med. 2022;28:378–87.

5. Ail D, Malki H, Zin EA, Dalkara D. Adeno-associated virus (AAV) - based gene therapies for retinal diseases: where are we? Appl Clin Genet. 2023;16:111–30.

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