Developing HDAC4-Selective Protein Degraders To Investigate the Role of HDAC4 in Huntington's Disease Pathology

J Med Chem. 2022 Sep 22;65(18):12445-12459. doi: 10.1021/acs.jmedchem.2c01149. Epub 2022 Sep 13.

Abstract

Huntington's disease (HD) is a lethal autosomal dominant neurodegenerative disorder resulting from a CAG repeat expansion in the huntingtin (HTT) gene. The product of translation of this gene is a highly aggregation-prone protein containing a polyglutamine tract >35 repeats (mHTT) that has been shown to colocalize with histone deacetylase 4 (HDAC4) in cytoplasmic inclusions in HD mouse models. Genetic reduction of HDAC4 in an HD mouse model resulted in delayed aggregation of mHTT, along with amelioration of neurological phenotypes and extended lifespan. To further investigate the role of HDAC4 in cellular models of HD, we have developed bifunctional degraders of the protein and report the first potent and selective degraders of HDAC4 that show an effect in multiple cell lines, including HD mouse model-derived cortical neurons. These degraders act via the ubiquitin-proteasomal pathway and selectively degrade HDAC4 over other class IIa HDAC isoforms (HDAC5, HDAC7, and HDAC9).

MeSH terms

  • Animals
  • Disease Models, Animal
  • Drug Development
  • Histone Deacetylases* / metabolism
  • Huntingtin Protein / genetics
  • Huntingtin Protein / metabolism
  • Huntington Disease* / genetics
  • Mice
  • Neurons / metabolism
  • Proteolysis
  • Ubiquitins

Substances

  • Huntingtin Protein
  • Ubiquitins
  • Hdac5 protein, mouse
  • Histone Deacetylases