Prof Sarah Tabrizi announces ASO huntingtin lowering trial for Huntington's disease
Channel: HD Buzz
Date: 2014-10-08

Prof Sarah Tabrizi of UCL, London, announces details of the first ever  trial of an agent designed to lower production of the mutant huntingtin protein that causes Huntington's disease, in partnership with Isis Pharmaceuticals. The trial is the first in HD patients to use 'huntingtin lowering' or 'gene silencing' technology. Patients will receive injections of antisense oligonucleotide (ASO) into the spinal fluid by lumbar puncture. Full details of the trial are awaited; it is expected to begin in the first half of 2015 at multiple sites.