Conference Coverage
First Clinical Trial of a Genetic Therapy for Huntington’s Disease Nears Completion
VANCOUVER—Antisense oligonucleotides hold promise as treatments for Huntington’s disease, according to an overview delivered at the 21st...
—Erik Greb
Suggested Reading
Aronin N, DiFiglia M. Huntingtin-lowering strategies in Huntington’s disease: antisense oligonucleotides, small RNAs, and gene editing. Mov Disord. 2014;29(11):1455-1461.
Kordasiewicz HB, Stanek LM, Wancewicz EV, et al. Sustained therapeutic reversal of Huntington’s disease by transient repression of huntingtin synthesis. Neuron. 2012;74(6):1031-1044.
Skotte NH, Southwell AL, Østergaard ME, et al. Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patients. PLoS One. 2014;9(9):e107434.
Wild EJ, Tabrizi SJ. Therapies targeting DNA and RNA in Huntington’s disease. Lancet Neurol. 2017;16(10):837-847.