Researchers use iPSCs to create a disease in a dish model of Huntington's disease in transgenic nonhuman primates.
A new study reports a potential new treatment for seizures which often occur in children with genetic metabolic disorders and people with liver failure.
Researchers find a single gene which encodes two separate proteins from the same sequence of messenger RNA. The finding could provide new strategies for treating SCA6.
Researchers identified 21 proteins that specifically bind to a protein called ataxin-1. Twelve of these proteins enhance the misfolding of ataxin-1 and thus promote the formation of harmful protein aggregate structures, whereas nine of them prevent the misfolding.
