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Drug targeting of dysregulated transcription in Huntington's disease

Aleksey G Kazantsev1, Steven M Hersch

  • 1Harvard Medical School, MassGeneral Institute for Neurodegenerative Disease, Massachusetts General Hospital, Charlestown, MA 02129-4404, USA. akazantsev@partners.org

Progress in Neurobiology
|March 24, 2007
PubMed

Insights

Huntington's disease (HD) involves widespread transcriptional dysregulation due to mutant huntingtin protein interactions. Current research reviews therapeutic strategies targeting these complex transcriptional pathways for HD treatment.

Area of Science:

  • Neuroscience
  • Genetics
  • Molecular Biology

Background:

  • Huntington's disease (HD) is characterized by significant transcriptional dysregulation.
  • The underlying cause involves aberrant protein-protein interactions between mutant huntingtin and transcription factors.
  • Identifying key transcriptional regulators in HD remains a challenge due to the widespread changes in RNA transcripts.

Purpose of the Study:

  • To review recent therapeutic advances targeting transcriptional deregulation in Huntington's disease.
  • To discuss potential drug discovery strategies for altered transcriptional pathways in HD.

Main Methods:

  • Review of current scientific literature on Huntington's disease therapeutics.
  • Analysis of challenges and opportunities in targeting transcriptional dysregulation.

Main Results:

  • Transcriptional dysregulation is a hallmark of HD, stemming from mutant huntingtin's interactions.
  • Developing targeted therapeutics is complex due to numerous altered RNA transcripts and non-conventional drug targets.
  • Progress has been made in developing therapies that address transcriptional alterations in HD.

Conclusions:

  • Targeting transcriptional deregulation offers a promising avenue for Huntington's disease therapeutics.
  • Further research into drug discovery strategies is needed to overcome challenges in targeting complex protein-protein interactions and widespread transcriptomic changes.

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