Blocking somatic repeat expansion and lowering huntingtin by RNAi synergize to attenuate Huntington’s disease pathogenesis in mice
Jillian Belgrad, Ashley Summers, Christian Landles, Jonathan R. Greene et autres
Huntington's disease (HD) is a progressive neurodegenerative disorder with no approved therapies. Despite multiple clinical trials, huntingtin (HTT)-lowering strategies have yet to show meaningful clinical benefit. Both somatic expansion and toxic HTT species are key molecular drivers of HD, yet therapeutic strategies …
us, gb (code pays fourni par la source)
0 citations
Science Translational Medicine