Small molecule correctors divert CFTR-F508del from ERAD by stabilizing sequential folding states

por Riepe, Celeste, Wąchalska, Magda, Deol, Kirandeep K., Amaya, Anais K., Porteus, Matthew H., Olzmann, James A., Kopito, Ron R.
Publicado 2023

Efficient in vivo editing of OTC-deficient patient-derived primary human hepatocytes

por Ginn, Samantha L., Amaya, Anais K., Liao, Sophia H.Y., Zhu, Erhua, Cunningham, Sharon C., Lee, Michael, Hallwirth, Claus V., Logan, Grant J., Tay, Szun S., Cesare, Anthony J., Pickett, Hilda A., Grompe, Markus, Dilworth, Kimberley, Lisowski, Leszek, Alexander, Ian E.
Publicado 2019

Novel human liver-tropic AAV variants define transferable domains that markedly enhance the human tropism of AAV7 and AAV8

por Cabanes-Creus, Marti, Navarro, Renina Gale, Zhu, Erhua, Baltazar, Grober, Liao, Sophia H.Y., Drouyer, Matthieu, Amaya, Anais K., Scott, Suzanne, Nguyen, Loan Hanh, Westhaus, Adrian, Hebben, Matthias, Wilson, Laurence O.W., Thrasher, Adrian J., Alexander, Ian E., Lisowski, Leszek
Publicado 2021

Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality

por Cabanes-Creus, Marti, Ginn, Samantha L., Amaya, Anais K., Liao, Sophia H.Y., Westhaus, Adrian, Hallwirth, Claus V., Wilmott, Patrick, Ward, Jason, Dilworth, Kimberley L., Santilli, Giorgia, Rybicki, Arkadiusz, Nakai, Hiroyuki, Thrasher, Adrian J., Filip, Adrian C., Alexander, Ian E., Lisowski, Leszek
Publicado 2018

Attenuation of Heparan Sulfate Proteoglycan Binding Enhances In Vivo Transduction of Human Primary Hepatocytes with AAV2

por Cabanes-Creus, Marti, Westhaus, Adrian, Navarro, Renina Gale, Baltazar, Grober, Zhu, Erhua, Amaya, Anais K., Liao, Sophia H.Y., Scott, Suzanne, Sallard, Erwan, Dilworth, Kimberley L., Rybicki, Arkadiusz, Drouyer, Matthieu, Hallwirth, Claus V., Bennett, Antonette, Santilli, Giorgia, Thrasher, Adrian J., Agbandje-McKenna, Mavis, Alexander, Ian E., Lisowski, Leszek
Publicado 2020

Assessment of Pre-Clinical Liver Models Based on Their Ability to Predict the Liver-Tropism of Adeno-Associated Virus Vectors

por Westhaus, Adrian, Cabanes-Creus, Marti, Dilworth, Kimberley L., Zhu, Erhua, Salas Gómez, David, Navarro, Renina G., Amaya, Anais K., Scott, Suzanne, Kwiatek, Magdalena, McCorkindale, Alexandra L., Hayman, Tara E., Frahm, Silke, Perocheau, Dany P., Tran, Bang Manh, Vincan, Elizabeth, Wong, Sharon L., Waters, Shafagh A., Riddiough, Georgina E., Perini, Marcos V., Wilson, Laurence O.W., Baruteau, Julien, Diecke, Sebastian, González-Aseguinolaza, Gloria, Santilli, Giorgia, Thrasher, Adrian J., Alexander, Ian E., Lisowski, Leszek
Publicado 2023