Ontology highlight
ABSTRACT:
SUBMITTER: Palfi S
PROVIDER: S-EPMC6157351 | biostudies-literature | 2018 Sep
REPOSITORIES: biostudies-literature
Palfi Stéphane S Gurruchaga Jean Marc JM Lepetit Hélène H Howard Katy K Ralph G Scott GS Mason Sarah S Gouello Gaëtane G Domenech Philippe P Buttery Philip C PC Hantraye Philippe P Tuckwell Nicola J NJ Barker Roger A RA Mitrophanous Kyriacos A KA
Human gene therapy. Clinical development 20180901 3
Parkinson's disease is typically treated with oral dopamine replacement therapies. However, long-term use is complicated by motor fluctuations from intermittent stimulation of dopamine receptors and off-target effects. ProSavin, a lentiviral vector based gene therapy that delivers local and continuous dopamine, was previously shown to be well tolerated in a Phase I/II first-in-human study, with significant improvements in motor behavior from baseline at 1 year. Here, patients with Parkinson's di ...[more]