1. Progress on stem cell research towards the treatment of Parkinson's disease
- Author
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Katya McDonagh, Stuart A J Gibson, Sanbing Shen, and Guo-Dong Gao
- Subjects
Pathology ,medicine.medical_specialty ,Parkinson's disease ,Somatic cell ,Genetic enhancement ,defined factors ,Induced Pluripotent Stem Cells ,Medicine (miscellaneous) ,Review ,Biology ,Regenerative Medicine ,Biochemistry, Genetics and Molecular Biology (miscellaneous) ,Regenerative medicine ,reprogramming factors ,alpha-synuclein expression ,medicine ,Animals ,Humans ,Induced pluripotent stem cell ,Embryonic Stem Cells ,Clinical Trials as Topic ,human fibroblasts ,human es ,rat model ,Dopaminergic Neurons ,functional dopaminergic-neurons ,Parkinson Disease ,Cell Biology ,Stem Cell Research ,medicine.disease ,graft-survival ,Embryonic stem cell ,Substantia Nigra ,Molecular Medicine ,nigral transplants ,Stem cell ,human ips cells ,Reprogramming ,Neuroscience - Abstract
Parkinson's disease (PD) is a neurodegenerative disorder characterized by the progressive accumulation of Lewy body inclusions along with selective destruction of dopaminergic (DA) neurons in the nigrostriatal tract of the brain. Genetic studies have revealed much about the pathophysiology of PD, enabling the identification of both biomarkers for diagnosis and genetic targets for therapeutic treatment, which are evolved in tandem with the development of stem cell technologies. The discovery of induced pluripotent stem (iPS) cells facilitates the derivation of stem cells from adult somatic cells for personalized treatment and thus overcomes not only the limited availability of human embryonic stem cells but also ethical concerns surrounding their use. Non-viral, non-integration, or non-DNA-mediated reprogramming technologies are being developed. Protocols for generating midbrain DA neurons are undergoing constant refinement. The iPS cell-derived DA neurons provide cellular models for investigating disease progression in vitro and for screening molecules of novel therapeutic potential and have beneficial effects on improving the behavior of parkinsonian animals. Further progress in the development of safer non-viral/non-biased reprogramming strategies and the subsequent generation of homogenous midbrain DA neurons shall pave the way for clinical trials. A combined approach of drugs, cell replacement, and gene therapy to stop disease progression and to improve treatment may soon be within our reach.
- Published
- 2012