Edited cells offer new hope for Parkinson’s treatment

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Parkinson’s is the fastest-growing neurological condition in the world, and currently, there is no cure. 1 in 37 people alive today in the UK will be diagnosed with Parkinson’s in their lifetime. However key advancements offer new hope as research can help future treatments of the condition.    

Parkinson’s disease is a progressive disorder that is caused by degeneration of nerve cells, these cells die or become impaired, losing the ability to produce an important chemical; dopamine. The absence of dopamine makes it hard for the brain to coordinate muscle movements, causing motor symptoms, such as tremors.

Clinical studies on cell replacement therapy have been trialled in the past and have shown that although some patients saw their motor symptoms improve, others had transplant-induced dyskinesias – abnormal, uncontrolled and involuntary movement. The replacement cells also acquired the hallmarks of Parkinson’s, known as Lewy bodies.

Professor Tilo Kunath, group leader at the Centre for Regenerative Medicine at the University of Edinburgh and team have used a gene-editing tool known as CRISPR-Cas9. This technique allows scientists to edit parts of the genome by removing, adding or altering specific sections of the DNA sequence. These new laboratory-generated midbrain dopaminergic (mDA) cells did not develop signs of the disease, Lewy bodies, unlike those that were not gene-edited.  

Transplantation of dopamine-producing neurons has proved useful in disease management because it can reinnervate Parkinson’s affected brain regions, restore dopamine levels and provide symptom relief. These advances could be most beneficial in young-onset Parkinson’s and younger patients.

This discovery has the potential to significantly affect the development of next-generation cell-based therapies. Clinical implications that they might be eventually considered as possible donor cells in future Parkinson’s trials.

The work was funded by the UK Centre for Mammalian Synthetic Biology, UCB and Cure Parkinson’s.

More details: Engineering synucleinopathy‐resistant human dopaminergic neurons by CRISPR‐mediated deletion of the SNCA gene - Chen - 2019 - European Journal of Neuroscience - Wiley Online Library