Correction of Familial Hypercholesterolaemia

Correction of Familial Hypercholesterolaemia

Correction of Familial Hypercholesterolaemia Development and rescue of human familial hypercholesterolaemia in a xenograft mouse model Bissig-Choisat et. al., 2015 – Study Summary Beatrice Bissig-Choisat, Lili Wang, Xavier Legras, Pradip K. Saha, Leon Chen,...
In Vivo AAV Selection

In Vivo AAV Selection

In Vivo AAV Selection Selection and evaluation of clinically relevant AAV variants in a xenograft liver model Lisowski et. al., 2014 – Study Summary Leszek Lisowski, Allison P. Dane, Kirk Chu, Yue Zhang, Sharon C. Cunningham, Elizabeth M. Wilson, Sean Nygaard,...
AAV Gene Repair of HT1

AAV Gene Repair of HT1

AAV Gene Repair of HT1 Adeno-Assocciated Virus Gene Repair Corrects a Mouse Model of Hereditary Tyrosinemia In Vivo Paulk et. al., 2010 – Study Summary Nicole K. Paulk,  Karsten Wursthorn,  Zhongya Wang,  Milton J. Finegold,  Mark A. Kay, and  Markus Grompe...
RNAi Prevents HBV Selection

RNAi Prevents HBV Selection

RNAi Prevents HBV Selection Combinatorial RNA Interference Therapy Prevents Selection of Preexisting HBV Variants in Human Liver Chimeric Mice Shih et. al., 2015 – Study Summary Yao-Ming Shih, Cheng-Pu Sun, Hui-Hsien Chou, Tzu-Hui Wu, Chun-Chi Chen, Ping-Yi Wu,...
Ex Vivo Correction of HT1

Ex Vivo Correction of HT1

Ex Vivo Correction of HT1 Curative ex vivo hepatocyte-directed gene editing in a mouse model of hereditary tyrosinemia type 1 VanLith et. al., 2018 – Study Summary Caitlin VanLith, Rebekah Guthman, Clara T. Nicolas, Kari Allen, Zeji Du, Dong Jin Joo, Scott L....