Article
Huntingtin and the Synapse
15 Jun 2021
Abstract excerpt
) gene, which results in the production of a pathogenic mutant HTT protein (mHTT). Although there is no cure at present for HD, a number of RNA-targeting therapies have recently entered clinical trials which aim to lower mHTT production through the use of antisense oligonucleotides (ASOs) and RNAi. However, many of these treatment strategies are non-selective in that they cannot differentiate between...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.