Lung SORT LNPs enable precise homology-directed repair mediated CRISPR/Cas genome correction in cystic fibrosis models

Approximately 10% of Cystic Fibrosis (CF) patients, particularly those with CF transmembrane conductance regulator (CFTR) gene nonsense mutations, lack effective treatments. The potential of gene correction therapy through delivery of the CRISPR/Cas system to CF-relevant organs/cells is hindered by...

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Detalles Bibliográficos
Autores principales: Wei, Tuo, Sun, Yehui, Cheng, Qiang, Chatterjee, Sumanta, Traylor, Zachary, Johnson, Lindsay T., Coquelin, Melissa L., Wang, Jialu, Torres, Michael J., Lian, Xizhen, Wang, Xu, Xiao, Yufen, Hodges, Craig A., Siegwart, Daniel J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10640563/
https://www.ncbi.nlm.nih.gov/pubmed/37951948
http://dx.doi.org/10.1038/s41467-023-42948-2

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10640563/
https://www.ncbi.nlm.nih.gov/pubmed/37951948
http://dx.doi.org/10.1038/s41467-023-42948-2

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