New approach may restore dopamine production in Parkinson's

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New approach may restore dopamine production in Parkinson's

Postby whbio » Apr 13 2017 6:08 pm

A team of scientists from Karolinska Institutet in Sweden, Medical University of Vienna in Austria, Malaga University in Spain, and Stanford University in the USA, has discovered a treatment approach for Parkinson's disease, a progressive neurodegenerative disease characterized by the loss of dopamine-producing neurons in the brain.

Over 1% of people over 65 will develop Parkinson’s disease, making it the second most prevalent neurodegenerative diseases. Disease symptoms vary from person to person and get worse over time. Typical symptoms include t[url][/url]remor or shaking, slowed movement, stiff muscles and achiness, difficulty with walking and balance, and speech changes. These symptoms are a consequence of degeneration of neurons in the brain that normally produce and release dopamine, a neurotransmitter crucial for movement, memory, attention, and many other functions.

One promising approach to treating Parkinson’s disease is cell replacement therapy. Previous studies regarding cell replacement therapy have mainly focused on transplantation of the cell types affected by the disease. For the new study, the investigators tested an alternative method in which existing brain cells are reprogrammed to take over the job of cells destroyed by the disease.

In the study, the researchers used a cocktail of small molecules to reprogramme human astrocytes in vitro, and mouse astrocytes in vivo. The cocktail includes three transcription factors, NEUROD1, ASCL1 and LMX1A, and the microRNA miR218. In vitro experiments showed that the cocktail caused human astrocytes to transform into a form that closely resembled dopamine neurons. After the researchers administrated the same cocktail to a mouse model of Parkinson’s disease, disease symptoms lessened.

Taken together, the results indicate that reprogramming existing brain cells into induced dopamine neurons might be an approach to treating Parkinson’s disease. Although more research is needed to confirm the safety and effectiveness of this approach, the potential of this type of cell replacement therapy is exciting. The findings were reported in Nature Biotechnology. (NEUROD1, ASCL1 and LMX1A, as well as related antibodies can be provided by Cusabio.)
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