Motor neurone disease
Motor neurone disease (MND), sometimes referred to as amyotrophic lateral sclerosis (ALS) , is the name given to a group of progressive neurological conditions where specific nerve cells, or neurons, don’t work normally. This leads to increasing physical disability, as muscles are unable to receive messages from the neurons to move or activate. Without these signals, muscle weakness, wasting and paralysis develop.
MND usually develops quickly once symptoms begin, with an average life expectancy of 2-3 years from diagnosis. It can affect anyone, at any age, but usually develops in people over the age of 40.
How The Florey is making a difference
There has been steady progress in identifying genetic factors that cause or predispose people to MND. This has resulted in a large number of genetically engineered laboratory models to test new treatment approaches for MND.
The Florey has an outstanding program of research in MND using patient-derived stem cell based and animal models. Our research using cutting-edge techniques spans key areas in MND, including protein folding, energy metabolism, support cells, immune cells, gene and stem cell therapy approaches.
- Defining unique molecular markers of upper motor neurons in MND
- Developing BBB-permeable ASO to target ataxin-2 and mitigate TDP-43 proteinopathies for amyotrophic lateral sclerosis therapy
- Developing SMN gene therapy for SMA and MND
- Development of peptide-oligonucleotide conjugates to target poly (ADP-ribose) polymerase for new RNA-based amyotrophic lateral sclerosis therapy
- Development of stem-cell based therapies for motor neurone disease
Latest Florey news on Motor neurone disease