Article
Reprogramming metabolic pathways in vivo with CRISPR/Cas9 genome editing to treat hereditary tyrosinaemia.
Nature communications - 30 Aug 2016
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
Abstract excerpt
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 in a disease-associated pathway to render the phenotype benign. Using CRISPR/Cas9 in vivo, we convert hepatocytes...
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