Brain Targeted AAV1-GALC Gene Therapy Reduces Psychosine and Extends Lifespan in a Mouse Model of Krabbe Disease

por Herdt, Aimee R., Peng, Hui, Dickson, Dennis W., Golde, Todd E., Eckman, Elizabeth A., Lee, Chris W.
Publicado 2023

Galactosylceramidase deficiency and pathological abnormalities in cerebral white matter of Krabbe disease

por Iacono, Diego, Koga, Shunsuke, Peng, Hui, Manavalan, Arulmani, Daiker, Jessica, Castanedes-Casey, Monica, Martin, Nicholas B., Herdt, Aimee R., Gelb, Michael H., Dickson, Dennis W., Lee, Chris W.
Publicado 2022