PROJECT 10:

Host:

Department of Medical and Molecular Genetics, King‘s College London

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PI:
Gillian Bates

Fellow:
Raffaella Tulino

Project 2

Huntington's disease (HD) is a neurodegenerative genetic disorder that affects muscle coordination and leads to cognitive decline and psychiatric problems. It is caused by a CAG repeat expansion in Exon 1 of the huntingtin (HTT) gene which translates to a polyglutamine tract close to the N-terminus of the huntingtin protein (HTT). Symptoms progress over a period of 15-20 years until death and disease modifying treatments do not currently exist.

The main focus of our work uses mouse models of HD to

  • Better understand the molecular pathogenesis of HD
  • Validate therapeutic targets
  • Participate in the preclinical development of therapeutic interventions