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J Parkinsons Dis. 2018;8(s1):S123-S130. doi: 10.3233/JPD-181485.

Repairing the Brain: Gene Therapy.

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Molecular Neuromodulation, Wallenberg Neuroscience Center, Lund University, Lund, Sweden.


In vivo gene therapy for neurodegenerative disorders has turned out to be a formidable challenge. It is a field not much older than twenty years, but we were many who would have predicted a much easier path towards the clinic using this treatment modality. For Parkinson's disease patients, this has meant a frustrating wait, seeing many promising therapies being forgotten after a few pre-clinical proof-of-concept studies. The reasons for this are both scientific and economical. However, this is slowly but surely changing and over the next two decades we will see a very exciting development in this field. In a foreseeable future, gene therapy will be a very natural component of many clinical therapies, not least in Parkinson's disease.


Genetic therapy; Parkinson’s disease; clinical trial; dependovirus; gene editing; neuroprotection; rejuvenation

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