Novel vectors and approaches for gene therapy in liver diseasesKey points

by Sheila Maestro, Nicholas D. Weber, Nerea Zabaleta, Rafael Aldabe, Gloria Gonzalez-Aseguinolaza
Published 2021-08-01

Gene therapy for progressive familial intrahepatic cholestasis type 3 in a clinically relevant mouse model

by Nicholas D. Weber, Leticia Odriozola, Javier Martínez-García, Veronica Ferrer, Anne Douar, Bernard Bénichou, Gloria González-Aseguinolaza, Cristian Smerdou
Published 2019-12-01

AAV-mediated delivery of zinc finger nucleases targeting hepatitis B virus inhibits active replication.

by Nicholas D Weber, Daniel Stone, Ruth Hall Sedlak, Harshana S De Silva Feelixge, Pavitra Roychoudhury, Joshua T Schiffer, Martine Aubert, Keith R Jerome
Published 2014-01-01

CRISPR-Cas9 gene editing of hepatitis B virus in chronically infected humanized mice

by Daniel Stone, Kelly R. Long, Michelle A. Loprieno, Harshana S. De Silva Feelixge, Elizabeth J. Kenkel, R. Matt Liley, Stephen Rapp, Pavitra Roychoudhury, Thuy Nguyen, Laurence Stensland, Rossana Colón-Thillet, Lindsay M. Klouser, Nicholas D. Weber, Connie Le, Jessica Wagoner, Erin A. Goecker, Alvason Zhenhua Li, Karsten Eichholz, Lawrence Corey, D. Lorne Tyrrell, Alexander L. Greninger, Meei-Li Huang, Stephen J. Polyak, Martine Aubert, John E. Sagartz, Keith R. Jerome
Published 2021-03-01