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Efficient suppression of endogenous CFTR nonsense mutations using anticodon-engineered transfer RNAs
Nonsense mutations or premature termination codons (PTCs) comprise ∼11% of all genetic lesions, which result in over 7,000 distinct genetic diseases. Due to their outsized impact on human health, considerable effort has been made to find therapies for nonsense-associated diseases. Suppressor tRNAs h...
| Autores principales: | , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
American Society of Gene & Cell Therapy
2022
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9126842/ https://www.ncbi.nlm.nih.gov/pubmed/35664697 http://dx.doi.org/10.1016/j.omtn.2022.04.033 |