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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Detalles Bibliográficos
Autores principales: Aaron, Ksenia A., Pekrun, Katja, Atkinson, Patrick J., Billings, Sara E., Abitbol, Julia M., Lee, Ina A., Eltawil, Yasmin, Chen, Yuan-Siao, Dong, Wuxing, Nelson, Rick F., Kay, Mark A., Cheng, Alan G.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10471831/
https://www.ncbi.nlm.nih.gov/pubmed/37663645
http://dx.doi.org/10.1016/j.omtm.2023.08.004

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