Selection of viral capsids and promoters affects the efficacy of rescue of Tmprss3-deficient cochlea

Adeno-associated virus (AAV)-mediated gene transfer has shown promise in rescuing mouse models of genetic hearing loss, but how viral capsid and promoter selection affects efficacy is poorly characterized. Here, we tested combinations of AAVs and promoters to deliver Tmprss3, mutations in which are...

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Bibliographic Details
Main Authors: Ksenia A. Aaron, Katja Pekrun, Patrick J. Atkinson, Sara E. Billings, Julia M. Abitbol, Ina A. Lee, Yasmin Eltawil, Yuan-Siao Chen, Wuxing Dong, Rick F. Nelson, Mark A. Kay, Alan G. Cheng
Format: Article
Language:English
Published: Elsevier 2023-09-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050123001183