In vivo correction of cystic fibrosis mediated by PNA nanoparticles

Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator (CFTR) gene. We sought to correct the multiple organ dysfunction of the F508del CF-causing mutation using systemic delivery of peptide nucleic acid gene editing technology mediated by biocompatible polymeric na...

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Detalles Bibliográficos
Autores principales: Piotrowski-Daspit, Alexandra S., Barone, Christina, Lin, Chun-Yu, Deng, Yanxiang, Wu, Douglas, Binns, Thomas C., Xu, Emily, Ricciardi, Adele S., Putman, Rachael, Garrison, Alannah, Nguyen, Richard, Gupta, Anisha, Fan, Rong, Glazer, Peter M., Saltzman, W. Mark, Egan, Marie E.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Association for the Advancement of Science 2022
Materias:
Biomedicine and Life Sciences
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9534507/
https://www.ncbi.nlm.nih.gov/pubmed/36197984
http://dx.doi.org/10.1126/sciadv.abo0522

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