mRNA and gene editing: Late breaking therapies in liver diseases

por Zabaleta, Nerea, Torella, Laura, Weber, Nicholas D., Gonzalez‐Aseguinolaza, Gloria
Publicado 2022

Novel vectors and approaches for gene therapy in liver diseases

por Maestro, Sheila, Weber, Nicholas D., Zabaleta, Nerea, Aldabe, Rafael, Gonzalez-Aseguinolaza, Gloria
Publicado 2021

Gene Therapy for Acquired and Genetic Cholestasis

por Martínez-García, Javier, Molina, Angie, González-Aseguinolaza, Gloria, Weber, Nicholas D., Smerdou, Cristian
Publicado 2022

A minimal bile salt excretory pump promoter allows bile acid-driven physiological regulation of transgene expression from a gene therapy vector

por Martínez-García, Javier, Molina, Manuela, Odriozola, Leticia, Molina, Angie, González-Aseguinolaza, Gloria, Weber, Nicholas D., Smerdou, Cristian
Publicado 2022

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

por Weber, Nicholas D., Odriozola, Leticia, Martínez-García, Javier, Ferrer, Veronica, Douar, Anne, Bénichou, Bernard, González-Aseguinolaza, Gloria, Smerdou, Cristian
Publicado 2019

AAV-Mediated Delivery of Zinc Finger Nucleases Targeting Hepatitis B Virus Inhibits Active Replication

por Weber, Nicholas D., Stone, Daniel, Sedlak, Ruth Hall, De Silva Feelixge, Harshana S., Roychoudhury, Pavitra, Schiffer, Joshua T., Aubert, Martine, Jerome, Keith R.
Publicado 2014

Use of an adeno‐associated virus serotype Anc80 to provide durable cure of phenylketonuria in a mouse model

por Kaiser, Robert A., Weber, Nicholas D., Trigueros‐Motos, Laia, Allen, Kari L., Martinez, Michael, Cao, William, VanLith, Caitlin J., Hillin, Lori G., Douar, Anne, González‐Aseguinolaza, Gloria, Aldabe, Rafael, Lillegard, Joseph B.
Publicado 2021

Digital detection of endonuclease mediated gene disruption in the HIV provirus

por Sedlak, Ruth Hall, Liang, Shu, Niyonzima, Nixon, De Silva Feelixge, Harshana S., Roychoudhury, Pavitra, Greninger, Alexander L., Weber, Nicholas D., Boissel, Sandrine, Scharenberg, Andrew M., Cheng, Anqi, Magaret, Amalia, Bumgarner, Roger, Stone, Daniel, Jerome, Keith R.
Publicado 2016

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

por Stone, Daniel, Long, Kelly R., Loprieno, Michelle A., De Silva Feelixge, Harshana S., Kenkel, Elizabeth J., Liley, R. Matt, Rapp, Stephen, Roychoudhury, Pavitra, Nguyen, Thuy, Stensland, Laurence, Colón-Thillet, Rossana, Klouser, Lindsay M., Weber, Nicholas D., Le, Connie, Wagoner, Jessica, Goecker, Erin A., Li, Alvason Zhenhua, Eichholz, Karsten, Corey, Lawrence, Tyrrell, D. Lorne, Greninger, Alexander L., Huang, Meei-Li, Polyak, Stephen J., Aubert, Martine, Sagartz, John E., Jerome, Keith R.
Publicado 2020