Back to top anchor

Cellular reprogramming: A unique approach to understanding Huntington's disease.

56 months
Approved budget:
Professor Bronwen Connor
Health issue:
Neurological (CNS)
Proposal type:
Lay summary
Huntington’s disease (HD) is a genetic neurological disorder that results in severe motor deficits due to loss of striatal medium spiny neurons (MSNs) in the brain. Inaccessibility to live human neurons is a major barrier to studying HD. To overcome this, we will use our novel reprogramming technology to turn skin cells from HD patients into immature brain cells (neural precursor cells) and subsequently mature MSNs displaying the genetic factors of HD. We will examine the molecular and cellular properties of HD-derived MSNs and determine their response to cell stress. Through an international collaboration, we will use our human cell model of HD to screen for novel drug-like compounds to prevent cell loss associated with HD pathology. This will provide a powerful system for understanding the cellular and molecular basis of HD, with potential to identify novel compounds for the treatment of HD that cannot be accomplished using current systems.