Steric Inhibition of 5′ UTR Regulatory Elements Results in Upregulation of Human CFTR

Cystic fibrosis (CF) is an autosomal recessive monogenic disease caused by mutations in the CFTR gene. Therapeutic approaches that are focused on correcting CFTR protein face the challenge of the heterogeneity in CFTR mutations and resulting defects. Thus, while several small molecules directed at C...

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Detalles Bibliográficos
Autores principales: Sasaki, Shruti, Sun, Rachel, Bui, Huynh-Hoa, Crosby, Jeff R., Monia, Brett P., Guo, Shuling
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2019
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6822282/
https://www.ncbi.nlm.nih.gov/pubmed/31351782
http://dx.doi.org/10.1016/j.ymthe.2019.06.016

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6822282/
https://www.ncbi.nlm.nih.gov/pubmed/31351782
http://dx.doi.org/10.1016/j.ymthe.2019.06.016

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