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expert reaction to an improved stem cell approach that could aid fight against Parkinson’s

Reactions to research published in European Journal of Neuroscience that describes a possible new treatment for Parkinson’s disease.

Dr Alena Pance, Senior Staff Scientist, Wellcome Trust Sanger Institute, said:

“The study presented by the Kunath group is yet another promising development of gene editing and stem cell technology for therapeutic applications. It shows that editing α-synuclein to prevent its expression renders cells resistant to the development of pathology marks characteristic of Parkinson’s disease. This has enormous potential to improve cell therapies by avoiding disease development in the transplanted cells and therefore provide long term benefits for the patients. Additionally, the use of stem cells differentiated in vitro can achieve pure dopaminergic neurone cultures, which could reduce graft-induced dyskinesias commonly observed in this type of therapy. Furthermore, the demonstration that the strategy also works with induced pluripotent cells (iPS) opens the possibility of patient-specific therapy (by using the patient’s own cells) which will increase compatibility and successful outcomes. It remains to be tested how will the engineered cells behave in vivo in terms of function and long term survival.”

Professor Ray Chaudhuri, Professor of Movement Disorders, Institute of Psychiatry, Psychology & Neuroscience, King’s College London, said:

“In Parkinson’s disease, stem cell therapy has been in the news now for many years and amidst many promises there have been major drawbacks. These include the formation of Lewy body in the grafted areas suggesting reactivation of the Parkinsonisan process and also chronic under-recognition from researchers and funders that Parkinson’s is not just a dopamine system disorder but a multi-neurotransmitter disorder where, apart from the typical visible symptoms of tremor and slowness and stooped postures, non-motor issues such as depression, sleep dysfunction, anxiety, fatigue and cognitive problems cause great distress to patients and carers. This new research, however, aims at a holistic and non-piecemeal approach to unravelling the process that leads to aggregation of alpha-synuclein, which is widely believed to be the key process underpinning the Parkinson’s symptoms. Using gene-editing processes and CRISPR they show that the grafted material could be resistant to attack by the Lewy body formation process. This work in mouse models represents a significant advance but the real challenge, as always, is to see how this technique can be translated to humans, what happens regarding tolerability and whether the effects also influence some key motor and non-motor symptoms. It is early days but this is indeed a significant advance.”

Professor Marios Politis, Professor of Neurology & Neuroimaging, Institute of Psychiatry, Psychology & Neuroscience, King’s College London, said:

“For over 30 years, cell replacement therapy has been investigated as a potential treatment for people suffering from Parkinson’s disease. Results so far have been inconsistent but there is hope that such therapies – once optimised – may provide solutions to alter the course of Parkinson’s disease. The work by Chen and colleagues is towards the correct direction of overcoming known barriers for the development of effective cell replacement therapies. However, there is more work to be done and I am looking forward to further developments to allow safe and effective translation of these and other important research developments in patients with Parkinson’s disease”

‘Engineering synucleinopathy-resistant human dopaminergic neurons by CRISPR-mediated deletion of the SNCA gene’ by Kunath et al. was published in European Journal of Neuroscience

The SMC also produced a Factsheet on genome editing which is available here:

Declared interests

Dr Pance: “I declare I have no vested interest in this research and have no connection to the research group involved.”

Prof Chaudhuri: “No interests”

Prof Polotis: “No interests to declare”

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