Queensland Institute of Medical Research, Griffith University and University of Queensland scientists researching a degenerative brain disease have developed a new way to understand its progression and safely test potential treatments.
The researchers have managed to generate Ataxia-telangiectasia (A-T) patients' nasal stem cells in the laboratory.
UQ Centre for Clinical Research Professor Martin Lavin, head of QIMR's Radiation Biology and Oncology Laboratory, said the breakthrough meant scientists could study exactly what was happening in a patient's brain.
And we think it means we can make a good contribution to coming up with a drug, to at least slow down the progress of the disease, Professor Lavin said.
Ataxia-telangiectasia (A-T) is an incurable, degenerative brain disease that leads to severe disabilities, a weakened immune system, and an increased risk of cancer.
Children who have the rare genetic condition are usually wheelchair-bound by their teens and rarely live beyond early adulthood.
Professor Lavin's team collaborated with Professor Alan Mackay-Sim from the Eskitis Institute for Drug Discovery at Griffith University to generate the stem cells from the olfactory organ in the nose, which are capable of changing into a wide range of specialised cell types.
These multipotent cells are known as olfactory neurosphere-derived (ONS) cells.
The ONS cells help scientists better understand how the condition develops, and allows them to screen a patient for any new drugs which might slow down the progression of the disease.
These cells from the nasal passage don't need to manipulated, or have viruses or genes added to them, they simply grow in the lab, Professor Lavin said.
The rest is here:
Queensland scientists provide insights into incurable brain disease