For the first time, scientists have created a human stem cell model of Parkinson’s disease in a petri dish.
Working at the New York Stem Cell Foundation (NYSCF) Research Institute, they took skin samples from a pair of identical twins, one of whom has Parkinson’s. They were then able to turn these into stem cells which they used to create working models in the lab.
Parkinson’s is a neurological condition that causes tremors, mobility problems, and pain, among other symptoms, and affects around 127,000 people in the UK. There are some treatments available but no cure and it usually gets worse over time. It’s linked to low levels of the pleasure hormone dopamine and a build-up of the protein α-synuclein.
It can be inherited but isn’t always – its exact cause is still a mystery. The chance of developing it is five times greater for people with a DNA mutation that triggers the production of an enzyme called glucocerebrosidase (GBA), but only 30% of people with the mutation develop Parkinson’s. Until now, scientists have had no way to study what other factors might be causing the disease – or to begin to develop potential cures.
Both the twins whose cells they’re studying carry the GBA mutation and when the researchers studied the cell models, they found that both twins had elevated α-synuclein and a reduced capacity to make and release dopamine, but this was more exaggerated in the neurons grown from the twin with Parkinson’s. His neurons also had more difficulty communicating with each other and had higher levels of an enzyme called monoamine oxidase B (MAO-B). The scientists believe this enzyme could give them a new option for developing more targeted treatments in future.
Dr Scott Noggle, NYSCF’s Vice President of Stem Cell Research said, ‘We hope our findings will be applicable to other Parkinson’s disease patients and other neurodegenerative disorders.’
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