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RNA Editing as a Therapeutic Approach for Retinal Gene Therapy Requiring Long Coding Sequences
RNA editing aims to treat genetic disease through altering gene expression at the transcript level. Pairing site-directed RNA-targeting mechanisms with engineered deaminase enzymes allows for the programmable correction of G>A and T>C mutations in RNA. This offers a promising therapeutic appro...
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| Опубликовано в: : | Int J Mol Sci |
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| Главные авторы: | , , , , |
| Формат: | Artigo |
| Язык: | Inglês |
| Опубликовано: |
MDPI
2020
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| Предметы: | |
| Online-ссылка: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7037314/ https://ncbi.nlm.nih.gov/pubmed/31991730 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms21030777 |
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