NeurosciencebioRxivPreprint
Delineating regional vulnerability in the neurodegenerative disease SCA1 using a conditional mutant ATXN1 mouse
Lisa A. Duvick, William M. Southern, Kellie A. Benzow…·Harry T. Orr
This study identifies brain regions and cell types contributing to SCA1 disease using conditional knockout mice, showing that targeting multiple areas and muscle is necessary for effective therapy.
2 citations