RNA Therapeutics Institute (@rti-umasschan.bsky.social)

Hot off the press from the RTI: Blocking somatic repeat expansion and lowering huntingtin via RNA interference synergize to prevent Huntington's disease pathogenesis in mice pubmed.ncbi.nlm.nih.gov/40667291/?ut... #RNA #RNATherapeutics

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Blocking somatic repeat expansion and lowering huntingtin via RNA interference synergize to prevent Huntington's disease pathogenesis in mice - PubMed Huntington's disease (HD) is a progressive neurodegenerative disorder with no approved therapies. Two major molecular drivers-somatic expansion of inherited CAG repeats and toxic mutant HTT (mHTT) variants-lead... https://pubmed.ncbi.nlm.nih.gov/40667291/?utm_source=Other&utm_medium=rss&utm_campaign=pubmed-2&utm_content=1hIuyCTdZ6aoVq_ZG2LVvryFp_CMOEco6wJSLrsxToxQKroQjW&fc=20250227163510&ff=20250729063939&v=2.18.0.post9+e462414