New Drug Technology Could Treat Huntington’s Disease

A first-in-man trial examined an antisense oligonucleotide intended to suppress huntingtin RNA.

Neurology Reviews. 2018 March;26(3):20

February 27, 2018|Neurology

—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.