期刊
JOURNAL OF PARKINSONS DISEASE
卷 11, 期 -, 页码 S135-S140出版社
IOS PRESS
DOI: 10.3233/JPD-212685
关键词
ATMP; clinical trial; dopaminergic neurons; regenerative therapy; stem cells; transplantation
After years of preclinical development, cell and gene therapies have become important products in Parkinson's disease clinical trials, with proven efficacy in preclinical models, but now need further validation of functionality and safety in clinical trials.
After many years of preclinical development, cell and gene therapies have advanced from research tools in the lab to clinical-grade products for patients, and today they constitute more than a quarter of all new Phase I clinical trials for Parkinson's disease. Whereas efficacy has been convincingly proven for many of these products in preclinical models, the field is now entering a new phase where the functionality and safety of these products will need to stand the test in clinical trials. If successful, these new products can have the potential to provide patients with a one-time administered treatment which may alleviate them from daily symptomatic dopaminergic medication.
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