Amino Acid Substitution at Phe80 of Mammalian Phenylalanine Hydroxylase Destabilizes Both Resting‐State and Activated Conformations Increasing the Population of Intermediates von Arturo, Emilia C., Merkel, George W., Borne, Elias, Hansen, Michael Riis, Lisowski, Sophia, Gupta, Kushol, Jaffe, Eileen K.

Volltext

Manipulation of a cation-π sandwich reveals conformational flexibility in phenylalanine hydroxylase von Arturo, Emilia C., Merkel, George W., Hansen, Michael R., Lisowski, Sophia, Almeida, Deeanne, Gupta, Kushol, Jaffe, Eileen K.

Volltext

Identification of liver-specific enhancer–promoter activity in the 3′ untranslated region of the wild-type AAV2 genome von Logan, Grant J, Dane, Allison P, Hallwirth, Claus V, Smyth, Christine M, Wilkie, Emilie E, Amaya, Anais K, Zhu, Erhua, Khandekar, Neeta, Ginn, Samantha L, Liao, Sophia H Y, Cunningham, Sharon C, Sasaki, Natsuki, Cabanes-Creus, Martí, Tam, Patrick P L, Russell, David W, Lisowski, Leszek, Alexander, Ian E

Volltext

Harnessing whole human liver ex situ normothermic perfusion for preclinical AAV vector evaluation von Cabanes-Creus, Marti, Liao, Sophia H. Y., Gale Navarro, Renina, Knight, Maddison, Nazareth, Deborah, Lau, Ngee-Soon, Ly, Mark, Zhu, Erhua, Roca-Pinilla, Ramon, Bugallo Delgado, Ricardo, Vicente, Ana F., Baltazar, Grober, Westhaus, Adrian, Merjane, Jessica, Crawford, Michael, McCaughan, Geoffrey W., Unzu, Carmen, González-Aseguinolaza, Gloria, Alexander, Ian E., Pulitano, Carlo, Lisowski, Leszek

Volltext

Characterization of the humanized FRG mouse model and development of an AAV-LK03 variant with improved liver lobular biodistribution von Cabanes-Creus, Marti, Navarro, Renina Gale, Liao, Sophia H.Y., Scott, Suzanne, Carlessi, Rodrigo, Roca-Pinilla, Ramon, Knight, Maddison, Baltazar, Grober, Zhu, Erhua, Jones, Matthew, Denisenko, Elena, Forrest, Alistair R.R., Alexander, Ian E., Tirnitz-Parker, Janina E.E., Lisowski, Leszek

Volltext

Development of CNS tropic AAV1-like variants with reduced liver-targeting following systemic administration in mice von Drouyer, Matthieu, Merjane, Jessica, Nazareth, Deborah, Knight, Maddison, Scott, Suzanne, Liao, Sophia H.Y., Ginn, Samantha L., Zhu, Erhua, Alexander, Ian E., Lisowski, Leszek

Volltext

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

Volltext

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

Volltext

Development of new adeno-associated virus capsid variants for targeted gene delivery to human cardiomyocytes von Kok, Cindy Y., Tsurusaki, Shinya, Cabanes-Creus, Marti, Igoor, Sindhu, Rao, Renuka, Skelton, Rhys, Liao, Sophia H.Y., Ginn, Samantha L., Knight, Maddison, Scott, Suzanne, Mietzsch, Mario, Fitzsimmons, Rebecca, Miller, Jessica, Mohamed, Tamer M.A., McKenna, Robert, Chong, James J.H., Hill, Adam P., Hudson, James E., Alexander, Ian E., Lisowski, Leszek, Kizana, Eddy

Volltext

Single amino acid insertion allows functional transduction of murine hepatocytes with human liver tropic AAV capsids von Cabanes-Creus, Marti, Navarro, Renina Gale, Liao, Sophia H.Y., Baltazar, Grober, Drouyer, Matthieu, Zhu, Erhua, Scott, Suzanne, Luong, Clement, Wilson, Laurence O.W., Alexander, Ian E., Lisowski, Leszek

Volltext

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

Volltext

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

Volltext