Potential disease modifying therapies for Huntington's disease, lessons learned and future opportunities
Publication details: 2022-07-01.Subject(s): Genre/Form: Online resources: Summary: Huntington's disease is the most frequent autosomal dominant neurodegenerative disorder, for which we have no approved disease-modifying treatments. The molecular pathogenesis of Huntington's disease is complex, with toxicity arising from full length expanded huntingtin and N-terminal fragments of huntingtin, which are both prone to misfolding due to proteolysis, from aberrant intron-1 splicing, and from somatic expansion of the CAG repeat in the HTT gene. Potential therapies for Huntington's disease include those targeting huntingtin DNA and RNA, clearance of huntingtin protein, DNA repair pathways, and other treatment strategies targeting inflammation and cell replacement. The early termination of the tominersen antisense oligonucleotide trials sent a strong signal that it is timely to reflect on lessons learned, where the field stands now, and our challenges and opportunities for the future./pmc/articles/PMC7613206/
/pubmed/35716694
Huntington's disease is the most frequent autosomal dominant neurodegenerative disorder, for which we have no approved disease-modifying treatments. The molecular pathogenesis of Huntington's disease is complex, with toxicity arising from full length expanded huntingtin and N-terminal fragments of huntingtin, which are both prone to misfolding due to proteolysis, from aberrant intron-1 splicing, and from somatic expansion of the CAG repeat in the HTT gene. Potential therapies for Huntington's disease include those targeting huntingtin DNA and RNA, clearance of huntingtin protein, DNA repair pathways, and other treatment strategies targeting inflammation and cell replacement. The early termination of the tominersen antisense oligonucleotide trials sent a strong signal that it is timely to reflect on lessons learned, where the field stands now, and our challenges and opportunities for the future.
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