Welsh Uni Scientists Join Brain Study Leap; SUCCESS IN WORLDWIDE RESEARCH ON HUNTINGTON'S
Byline: JULIA MCWATT
HUMAN brain cells showing aspects of Huntington's disease have been developed, opening up new research pathways for treating the fatal disorder.
An international consortium, including scientists from Cardiff University School of Bioscience, has taken cells from Huntington's disease patients and generated human brain cells that develop aspects of the disease in the laboratory.
The cells and the new technology will speed up research into understanding the disease and also accelerate drug discovery programmes aimed at treating this terminal genetic disorder.
Huntington's disease is an aggressive, neuro-degenerative disorder which causes loss of co-ordination, psychiatric problems, dementia and death. The physical symptoms of the condition can begin at any age, but usually begin between the ages of 35 and 44.
There is no effective treatment for the condition, caused by a toxic protein produced by a single defective gene, and there are around 6,700 people in the UK affected by the disease. Scientists have known the genetic cause for more than 20 years but research has been hampered by the lack of human brain cells with which to study the disease and screen for effective drugs.
The new breakthrough involves taking skin cells from patients with Huntington's disease. The scientific team reprogrammed these cells into stem cells which were then turned into the brain cells affected by the disorder.
The brain cells demonstrate characteristics of the disease and will allow the consortium to investigate the mechanisms that cause the brain cells to die.
Dr Nicholas Allen, one of the lead investigators at Cardiff, said: "This breakthrough allows us to generate brain cells with many of the hallmarks of this disease, within just a few weeks. …