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

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

doi:10.1038/ncomms12642

Nature Communications (Aug 2016)

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

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

Affiliations

Francis P. Pankowicz: Center for Cell and Gene Therapy, Baylor College of Medicine
Mercedes Barzi: Center for Cell and Gene Therapy, Baylor College of Medicine
Xavier Legras: Center for Cell and Gene Therapy, Baylor College of Medicine
Leroy Hubert: Department of Molecular and Human Genetics, Baylor College of Medicine
Tian Mi: Department of Pediatrics, Texas Children’s Hospital
Julie A. Tomolonis: Graduate Program in Translational Biology and Molecular Medicine, Baylor College of Medicine
Milan Ravishankar: Center for Cell and Gene Therapy, Baylor College of Medicine
Qin Sun: Department of Molecular and Human Genetics, Baylor College of Medicine
Diane Yang: Center for Cell and Gene Therapy, Baylor College of Medicine
Malgorzata Borowiak: Center for Cell and Gene Therapy, Baylor College of Medicine
Pavel Sumazin: Department of Pediatrics, Texas Children’s Hospital
Sarah H. Elsea: Department of Molecular and Human Genetics, Baylor College of Medicine
Beatrice Bissig-Choisat: Center for Cell and Gene Therapy, Baylor College of Medicine
Karl-Dimiter Bissig: Center for Cell and Gene Therapy, Baylor College of Medicine

DOI: https://doi.org/10.1038/ncomms12642
Journal volume & issue: Vol. 7, no. 1
pp. 1 – 6

Abstract

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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 hepatic tyrosine catabolism.

Published in Nature Communications

ISSN: 2041-1723 (Online)
Publisher: Nature Portfolio
Country of publisher: United Kingdom
LCC subjects: Science
Website: https://www.nature.com/ncomms/

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