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Identification of computationally designed skeletal and cardiac promoters with high specificity across AAV delivery routes

Abstract Background Precise control of transgene expression is essential for safe and effective AAV gene therapies. While capsid engineering has advanced tissue targeting, progress in developing regulatory elements that confine expression to specific tissues has lagged. This gap limits the ability t...

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Principais autores: Nicole Zielinska, Erin L. Howard, Brenna A. Y. Stevens, Melanie M. Goens, Cici Yang, Elena S. B. Campbell, Madison E. Hughes, Yanlong Pei, Dinghai Zheng, D. Benjamin Gordon, Alec A. K. Nielsen, Raja R. Srinivas, Jeff L. Caswell, Luis G. Arroyo, Sarah K. Wootton
Formato: Artigo
Idioma:Inglês
Publicado: BMC 2026-03-01
Series:BMC Biotechnology
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Acceso en liña:https://doi.org/10.1186/s12896-026-01141-1
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