In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery

The blood disorder, β-thalassaemia, is considered an attractive target for gene correction. Site-specific triplex formation has been shown to induce DNA repair and thereby catalyse genome editing. Here we report that triplex-forming peptide nucleic acids (PNAs) substituted at the γ position plus sti...

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Udgivet i:Nat Commun
Main Authors: Bahal, Raman, Ali McNeer, Nicole, Quijano, Elias, Liu, Yanfeng, Sulkowski, Parker, Turchick, Audrey, Lu, Yi-Chien, Bhunia, Dinesh C., Manna, Arunava, Greiner, Dale L., Brehm, Michael A., Cheng, Christopher J., López-Giráldez, Francesc, Ricciardi, Adele, Beloor, Jagadish, Krause, Diane S., Kumar, Priti, Gallagher, Patrick G., Braddock, Demetrios T., Mark Saltzman, W., Ly, Danith H., Glazer, Peter M.
Format: Artigo
Sprog:Inglês
Udgivet: Nature Publishing Group 2016
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5095181/
https://ncbi.nlm.nih.gov/pubmed/27782131
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/ncomms13304
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