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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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Автори: 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
Формат: Artigo
Мова:Inglês
Опубліковано: BMC 2026-03-01
Серія:BMC Biotechnology
Предмети:
AAV
Tissue-specific
Promoters
Algorithm
Targeted
Cardiac muscle
Онлайн доступ:https://doi.org/10.1186/s12896-026-01141-1
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