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Effective and Accurate Gene Silencing by a Recombinant AAV-Compatible MicroRNA Scaffold

Short hairpin RNAs that are delivered by recombinant adeno-associated virus (rAAV) have the potential to elicit long-term RNAi therapy for human disease. However, the discovery that short hairpin sequences can cause truncation of the rAAV genome calls into question the efficiency and gene-silencing...

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Dettagli Bibliografici
Pubblicato in:Mol Ther
Autori principali: Xie, Jun, Tai, Phillip W.L., Brown, Alexander, Gong, Shoufang, Zhu, Sha, Wang, Yi, Li, Chengjian, Colpan, Cansu, Su, Qin, He, Ran, Ma, Hong, Li, Jia, Ye, Hanqing, Ko, Jihye, Zamore, Phillip D., Gao, Guangping
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Gene & Cell Therapy 2020
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC7001000/
https://ncbi.nlm.nih.gov/pubmed/31843447
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2019.11.018
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