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

Hematopoietic stem cell transplantation (HSCT) is the only approved treatment for presymptomatic infantile globoid cell leukodystrophy (GLD [Krabbe disease]). However, correction of disease is not complete, and outcomes remain poor. Herein we evaluated HSCT, intravenous (IV) adeno-associated virus r...

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Veröffentlicht in:	Molecular therapy 2024-01, Vol.32 (1), p.44-58
Hauptverfasser:	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
Format:	Artikel
Sprache:	eng
Schlagworte:	AAV gene therapy Animals canine model combination therapy Disease Models, Animal Dogs Galactosylceramidase - genetics Genetic Therapy - methods hematopoietic stem cell transplantation Hematopoietic Stem Cell Transplantation - methods Krabbe disease leukodystrophy Leukodystrophy, Globoid Cell - genetics Leukodystrophy, Globoid Cell - therapy lysosomal storage disorder Psychosine
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