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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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| Pubblicato in: | Mol Ther |
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| Autori principali: | , , , , , , , , , , , , , , , |
| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
American Society of Gene & Cell Therapy
2020
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| Soggetti: | |
| 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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