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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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:EN
Published: Nature Portfolio 2016
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Science
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Online Access:https://doaj.org/article/c9a870f56fb24cb1bba81c0bca196a4d
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https://doaj.org/article/c9a870f56fb24cb1bba81c0bca196a4d

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