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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...
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 |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
2019
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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 |
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