Gene therapy for Parkinson disease—a hope, or a dream?
Author:
Publisher
Springer Science and Business Media LLC
Subject
Cellular and Molecular Neuroscience,Clinical Neurology
Link
http://www.nature.com/articles/nrneurol.2014.45.pdf
Reference10 articles.
1. Palfi, S. et al. Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial. Lancet http://dx.doi.org/10.1016/S0140-6736(13)61939-X .
2. Olanow, C. W., Obeso, J. A. & Stocchi, F. Continuous dopamine-receptor treatment of Parkinson's disease: scientific rationale and clinical implications. Lancet Neurol. 5, 677–687 (2006).
3. Marks, W. et al. Double-blind, sham-surgery controlled trial of gene delivery of AAV2–neurturin for Parkinson's Disease. Lancet Neurol. 9, 1164–1172 (2010).
4. Bartus, R. T. et al. Bioactivity of AAV2–neurturin gene therapy (CERE-120) in Parkinson's disease: a comparison of post-mortem and nonhuman primate brain tissue. Mov. Disord. 26, 27–36 (2011).
5. Decressac, M. et al. α-synuclein-induced down-regulation of Nurr1 disrupts GDNF signaling in nigral dopamine neurons. Sci. Transl. Med. 4, 163ra156 (2012).
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