Combination HSCT and intravenous AAV-mediated gene therapy in a canine model proves pivotal for translation of Krabbe disease therapy

Authors: Bradbury, Allison M., Bagel, Jessica, Swain, Gary, Miyadera, Keiko, Pesayco, Jill P., Assenmacher, Charles-Antoine, Brisson, Becky, Hendricks, Ian, Wang, Xiao H., Herbst, Zachary, Pyne, Nettie, Odonnell, Patricia, Shelton, G. Diane, Gelb, Michael, Hackett, Neil, Szabolcs, Paul, Vite, Charles H., Escolar, Maria

Source: In Molecular Therapy 3 January 2024 32(1):44-58

Disease correction in mucopolysaccharidosis type IIIB mice by intraparenchymal or cisternal delivery of a capsid modified AAV8 codon-optimized NAGLU vector.

Authors: Rouse, Courtney J, Hawkins, Kimberley, Kabbej, Nadia, Dalugdug, Justin, Kunta, Aishwarya, Kim, Mi-Jung, Someya, Shinichi, Herbst, Zachary, Gelb, Michael, Dinelli, Isabella, Butterworth, Elizabeth, Falk, Darin J, Rosenkrantz, Erinn, Elmohd, Hamza, Khaledi, Hamid, Mowafy, Samar, Ashby, Frederick, Heldermon, Coy D

Source: Human Molecular Genetics; Feb2023, Vol. 32 Issue 3, p417-430, 14p