Publication Date:
2015-04-29
Description:
Article Cystic fibrosis is a lethal genetic disorder commonly caused by the F508del mutation which is not amenable to gene therapy. Here, the authors use triplex-forming PNA molecules and donor DNA in biodegradable polymer nanoparticles to correct F508del and achieve clinically relevant levels of gene editing. Nature Communications doi: 10.1038/ncomms7952 Authors: Nicole Ali McNeer, Kavitha Anandalingam, Rachel J. Fields, Christina Caputo, Sascha Kopic, Anisha Gupta, Elias Quijano, Lee Polikoff, Yong Kong, Raman Bahal, John P. Geibel, Peter M. Glazer, W. Mark Saltzman, Marie E. Egan
Electronic ISSN:
2041-1723
Topics:
Biology
,
Chemistry and Pharmacology
,
Natural Sciences in General
,
Physics
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