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Engineering neurons for Parkinson's disease

Nature Biotechnology volume 17, pages 635–636 (1999)Cite this article

Restoring brain function by cell replacement is a new therapeutic strategy with great potential for treating human neurodegenerative disorders. Trials in patients with Parkinson's disease (PD) have demonstrated that the basic principle works; that is, functional deficits in the human brain caused by neuronal death can be reversed by transplantation of embryonic neurons1. However, a major obstacle preventing the widespread use of this approach in patients is the need for large amounts of human embryonic tissue. In this issue, Ernest Arenas and colleagues describe the first procedure based on cell engineering that could make it possible to generate unlimited numbers of neurons suitable for transplantation in PD2.

Parkinson's disease is the second most common neurodegenerative disorder, characterized by tremor, rigidity, and hypokinesia. l-dopa treatment is effective in the initial phase, but most patients eventually develop fluctuating therapeutic responses. The degeneration of the substantia nigra in PD leads to a loss of dopaminergic innervation of the striatum. Studies of PD patients prompted by successes in animal models have shown that intrastriatal grafts of human embryonic mesencephalic tissue can survive, leading to dopaminergic reinnervation of the host striatum—and clinical improvements1. In the most successful cases, engrafted patients have been able to withdraw from l-dopa treatment.

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Figure 1: Schematic of the engineering of dopamine neurons for grafting.

References

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  1. professor at the Section of Restorative Neurology, Wallenberg Neuroscience Center, University Hospital, 85 Lund, S-221, Sweden

    Olle Lindvall

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  1. Olle Lindvall
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Lindvall, O. Engineering neurons for Parkinson's disease. Nat Biotechnol 17, 635–636 (1999). https://doi.org/10.1038/10844

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