Reprogramming metabolic pathways in vivo with CRISPR/Cas9 genome editing to treat hereditary tyrosinaemia

Many metabolic liver disorders are refractory to drug therapy and require orthotopic liver transplantation. Here we demonstrate a new strategy, which we call metabolic pathway reprogramming, to treat hereditary tyrosinaemia type I in mice; rather than edit the disease-causing gene, we delete a gene...

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Publicat a:Nat Commun
Autors principals: Pankowicz, Francis P., Barzi, Mercedes, Legras, Xavier, Hubert, Leroy, Mi, Tian, Tomolonis, Julie A., Ravishankar, Milan, Sun, Qin, Yang, Diane, Borowiak, Malgorzata, Sumazin, Pavel, Elsea, Sarah H., Bissig-Choisat, Beatrice, Bissig, Karl-Dimiter
Format: Artigo
Idioma:Inglês
Publicat: Nature Publishing Group 2016
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Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC5013601/
https://ncbi.nlm.nih.gov/pubmed/27572891
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/ncomms12642
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