Huntington's disease gene therapy and nanomedicines may be available shortly

Although Huntington's disease-based gene mutation has long been established, pathophysiology from mutant gene to aberrant aggregation, neurotoxicity, metabolic dysfunction, and neuroimmunological dysfunction is convoluted and incomplete. We investigated innovative disease-modifying drugs that target various established pathogenic stages, from gene to RNA to protein pathways. Several of these medicines are being explored in human clinical trials, while others promise preclinical findings in vivo. The ultimate goal of these new treatments is to boost survival, function and quality of life and perhaps cure Huntington's disease. Gene therapies have tremendous potential to correct or alter the underlying defective trinucleotide expansion of DNA, which would prevent all erroneous downstream processes. In early pathogenic intervention, RNA-focused treatments also promise to avoid downstream HTT toxicity activation. For those targeting DNA and RNA levels, invasive administration (often necessitating direct intraparenychmal or intrathecal distribution) and potential off-target effects with accidental downregulation of non-HD-related genes or transcripts are typical treatment restrictions. Downstream therapies that are important include those that reduce MSN atrophy and neuroinflammation.Other novel preclinical development procedures include the use of endogenous, adult glial cells to repair striatal neurons and mutant HTT protein immunization. These drugs may be particularly relevant for usage in people with advanced disease if DNA or RNA-targeted therapy can not reverse previous neurotoxicity. These unique and new therapy approaches have generated hopes that Huntington's disease-modifying medicines may soon become reality.