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A reversible RNA on-switch that controls gene expression of AAV-delivered therapeutics in vivo

Widespread use of gene therapy technologies is limited in part by the lack of small genetic switches with wide dynamic ranges that control transgene expression without the requirement of additional protein components(1–5). Here, we engineered a class of type III hammerhead ribozymes to develop RNA s...

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Detalles Bibliográficos
Autores principales: Zhong, Guocai, Wang, Haimin, He, Wenhui, Li, Yujun, Mou, Huihui, Tickner, Zachary J., Tran, Mai H., Ou, Tianling, Yin, Yiming, Diao, Huitian, Farzan, Michael
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7008088/
https://www.ncbi.nlm.nih.gov/pubmed/31873216
http://dx.doi.org/10.1038/s41587-019-0357-y