New England Journal of Medicine
Targeting Huntingtin Expression in Patients with Huntington’s Disease
What this article backs
- Htt Gene Mutation enables Mutant Huntingtin Protein.
- Antisense Oligonucleotide decreases Mutant Huntingtin Protein.
In its own words
HTT Rx treatment resulted in a dose-dependent reduction in the concentration of mutant huntingtin in CSF (mean percentage change from baseline... -20%, -25%, -28%, -42%, and -38% in the HTT Rx 10-mg, 30-mg, 60-mg, 90-mg, and 120-mg dose groups
Huntington's disease is an autosomal-dominant neurodegenerative disease caused by CAG trinucleotide repeat expansion in HTT, resulting in a mutant huntingtin protein.
