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Gene therapy progress for Parkinson's disease
An early-stage clinical trial of gene therapy for Parkinson’s disease (PD) has reported promising results at the American Society of Gene and Cell Therapy (ASGCT) conference.
Oxford BioMedica’s ProSavin, which is injected directly to the brain, delivers genes that direct production of the neurotransmitter dopamine; PD is caused by the progressive loss of dopamine-producing cells in the brain, resulting in impaired control of movements. The therapeutic produced an average reduction of 43% in symptoms of shaking and stiffness for the nine patients in the trial, with no serious side-effects reported.
Gene therapies no longer excite media interest in the way they once did; there was perhaps a general disappointment that effective delivery and sustained expression of therapeutic genes proved to be a lot more difficult than originally envisaged. However, applications for a range of relatively common conditions such as PD and forms of cancer, as well as rare genetic disorders, are steadily progressing towards clinical application. It isn’t a quick fix, but for serious conditions gene therapy may offer real hope.