Lowering expression of the mutated Huntington Disease (HD) gene (HTT)    currently requires chronic administration of anti-sense oligonucleotides (ASO) or small interfering RNA (siRNA) into the cerebrospinal fluid (CSF) by intrathecal infusions or direct intracerebral injections. Ongoing clinical trials have demonstrated that intrathecal administration of anti-HTT ASO is well-tolerated for at least several years.  However, repetitive long-term intrathecal infusions can result in range of complications and may not be acceptable for a lifetime of  treatment.  In addition, treatment of patients with a greater disease burden (older subjects with high CAG repeat length) did not appear to impact clinical measures of progression.

Juan Sanchez-Ramos currently works at the Department of Neurology, University of South Florida. Juan does research in Neuroscience and Biology. Their most recent publication is 'Chitosan-Mangafodipir nanoparticles designed for intranasal delivery of siRNA and DNA to brain'.