Award

Therapeutic pathway reprogramming for metabolic liver disease

Description: PROJECT NARRATIVE We recently pioneered a novel therapeutic concept for metabolic disease, metabolic pathway reprogramming, in which we reroute a metabolic pathway to a benign disease phenotype using genome editing tools. Using tyrosinemia type I as a model metabolic disorder, we will address the translational challenges of this genome editing approach in a murine disease model and human liver chimeric mice.
Funding type: research
Funder scheme: R56
Start date: 2017-09-13
End date: 2018-09-12
Investigator: KARL-DIMITER BISSIG
Institution: BAYLOR COLLEGE OF MEDICINE
Funded outputs count: 10
Funder award ID: R56DK115461
OpenAlex ID: G6631083644
2025 · Marco De Giorgi, So Hyun Park, et al. · Science Translational Medicine · 8
2024 · Ilayda Ates, Callie Stuart, et al. · Hepatology Communications · 5
2023 · Mercedes Barzi, Collin G. Johnson, et al. · Science Translational Medicine · 26
2021 · Marco De Giorgi, Ang Li, et al. · Molecular Therapy — Methods & Clinical Development · 28
2020 · Renata Stripecke, Christian Münz, et al. · EMBO Molecular Medicine · 137
2018 · Francis P. Pankowicz, Mercedes Barzi, et al. · Gastroenterology · 22
2018 · Karl-Dimiter Bissig, Weiguo Han, et al. · Drug Metabolism and Disposition · 59
2018 · Jordan Kho, Xiaoyu Tian, et al. · The American Journal of Human Genetics · 55
2018 · Robert L. Kruse, Thomas Shum, et al. · Cytotherapy · 100
2017 · Robert L. Kruse, Thomas Shum, et al. · Molecular Therapy — Methods & Clinical Development · 19
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