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

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

Hereditary tyrosinaemia type I is caused by a gene defect that leads to a lethal accumulation of toxic metabolites in the liver. Here the authors use CRISPR/Cas9 to 'cure' the disease in mice by inactivating another gene, rather than targeting the disease-causing gene itself, to reroute he...

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Bibliographic Details
Main Authors: Francis P. Pankowicz, Mercedes Barzi, Xavier Legras, Leroy Hubert, Tian Mi, Julie A. Tomolonis, Milan Ravishankar, Qin Sun, Diane Yang, Malgorzata Borowiak, Pavel Sumazin, Sarah H. Elsea, Beatrice Bissig-Choisat, Karl-Dimiter Bissig
Format: Article
Language:English
Published: Nature Publishing Group 2016-08-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/ncomms12642

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https://doi.org/10.1038/ncomms12642

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