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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...
Autores principales: | 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 |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group
2016
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5013601/ https://www.ncbi.nlm.nih.gov/pubmed/27572891 http://dx.doi.org/10.1038/ncomms12642 |
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