Abstract
The second most common neurodegenerative disease, Parkinson’s disease (PD) is characterized by progressive loss of dopaminergic neurons which in turn causes the occurrence of several motor symptoms.
Current therapeutic options for PD are available to provide relief in primary motor symptoms, but their long-term effectiveness is limited. For this reason, alternative therapeutic options are being required in the form of stem cell and gene-based therapies. Several open-label clinical trials like intrastriatal transplantation of human fetal mesencephalic tissue have provided proof of principle that stem cell replacement therapy may provide significant relief to motor impairment in some PD patients, particularly diagnosed at early stage of disease onset. Today, stem cell-based therapies utilizing different mechanisms are being investigated to provide alternative treatment options. These therapies include cell replacement therapy itself, but also involve immunomodulatory and trophic actions to help promote intrinsic repair processes in damaged areas.
Furthermore, promising approaches based on gene therapy are also being developed for the cure of PD patients, some of which have already entered in clinical phases of their development. These clinical trials are mainly based on the treatment of motor symptoms (by modulating activity of the basal ganglia or by the introduction of genes involved in dopamine synthesis) or increasing dopaminergic neuron survival by transducing genes for neurotrophic factors.
In this chapter, we summarize current studies involving the use of stem cell and gene-based treatment for PD management. Here we provide an overview of currently available different types of stem cells and the different strategies of gene therapy that are being developed to treat PD patients.
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Acknowledgements
The authors wish to thank members of their laboratory for their research work and fruitful discussions. Research at the authors’ laboratory was funded by the MICINN-ISCIII (PI-10/00291 and MPY1412/09), MINECO (SAF2015-71140-R), and Comunidad de Madrid (NEUROSTEMCM consortium; S2010/BMD-2336).
Author Disclosure Statement: The authors declare that they have no competing interests.
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Palmer, C., Coronel, R., Bernabeu-Zornoza, A., Liste, I. (2019). Therapeutic Application of Stem Cell and Gene Therapy in Parkinson’s Disease. In: Singh, S., Joshi, N. (eds) Pathology, Prevention and Therapeutics of Neurodegenerative Disease. Springer, Singapore. https://doi.org/10.1007/978-981-13-0944-1_14
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DOI: https://doi.org/10.1007/978-981-13-0944-1_14
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