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

by 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
Published 2016

Development and rescue of human familial hypercholesterolaemia in a xenograft mouse model

by Bissig-Choisat, Beatrice, Wang, Lili, Legras, Xavier, Saha, Pradip K., Chen, Leon, Bell, Peter, Pankowicz, Francis P., Hill, Matthew C., Barzi, Mercedes, Leyton, Claudia Kettlun, Leung, Hon-Chiu Eastwood, Kruse, Robert L., Himes, Ryan W., Goss, John A., Wilson, James M., Chan, Lawrence, Lagor, William R., Bissig, Karl-Dimiter
Published 2015