Human brain development and function
The human nervous system is one of the most complex structures evolved to date. In order to understand how it functions, and dysfunctions in a diseased state, it is fundamental to decipher how it creates neuronal populations that form functional networks. Our research uses human stem cells as in vitro models to recapitulate key events of human brain development including induction of region-specific neural progenitors and terminally differentiated neurons and glia in order to better understand the cellular and molecular networks underlying central nervous system disorders.
Modelling ‘pleasure and pain’ in the dish using stem cells
Dorsal root ganglia (DRG) sensory neurons enable us to sense temperature, pressure, position, touch and pain. Not surprisingly, there are vast ranges of diseases and conditions, usually progressive, which can cause DRG neuropathies. Our research aims to develop efficient systems to derive sensory neurons from human pluripotent stem cells to further study their functional properties and develop regenerative therapies to treating peripheral neuropathies, such chronic pain.
Developing treatments for Friedreich Ataxia
Friedreich ataxia (FRDA) is an autosomal recessive disease characterised by neurodegeneration and cardiomyopathy. The cause of FRDA is due to mutations in the FXN gene, resulting in an insufficiency of the mitochondrial protein, Frataxin. Reduced levels of Frataxin protein leads to mitochondrial dysfunction, cell toxicity and cell death, particularly within the sensory nervous system and cardiac tissue. Our research is based on using stem cells derived from FRDA patients to develop therapies to treat the progressive neurodegeneration occurring in sensory neurons.