Systemic gene therapy for methylmalonic acidemia using the novel adeno-associated viral vector 44.9

por Chandler, Randy J., Di Pasquale, Giovanni, Sloan, Jennifer L., McCoy, Samantha, Hubbard, Brandon T., Kilts, Tina M., Manoli, Irini, Chiorini, John A., Venditti, Charles P.
Publicado 2022

Promoterless, Nuclease‐Free Genome Editing Confers a Growth Advantage for Corrected Hepatocytes in Mice With Methylmalonic Acidemia

por Chandler, Randy J., Venturoni, Leah E., Liao, Jing, Hubbard, Brandon T., Schneller, Jessica L., Hoffmann, Victoria, Gordo, Susana, Zang, Shengwen, Ko, Chih‐Wei, Chau, Nelson, Chiang, Kyle, Kay, Mark A., Barzel, Adi, Venditti, Charles P.
Publicado 2021

Metformin, phenformin, and galegine inhibit complex IV activity and reduce glycerol-derived gluconeogenesis

por LaMoia, Traci E., Butrico, Gina M., Kalpage, Hasini A., Goedeke, Leigh, Hubbard, Brandon T., Vatner, Daniel F., Gaspar, Rafael C., Zhang, Xian-Man, Cline, Gary W., Nakahara, Keita, Woo, Seungwan, Shimada, Atsuhiro, Hüttemann, Maik, Shulman, Gerald I.
Publicado 2022