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...

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Detalles Bibliográficos
Autores principales: Ko, Wooree, Porter, Joseph J., Sipple, Matthew T., Edwards, Katherine M., Lueck, John D.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
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

Internet

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

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