Antibiotic is gene therapy 'off' switch
Posted on: Wednesday, 2 September 2009, 13:39 CDT
University of Florida scientists say they've found a common antibiotic can become an off switch
for a gene therapy being developed for Parkinson's disease.
The scientists, led by Professor Ronald Mandel, said their discovery made during a study involving rats answers an important question -- how can new, therapeutic genes that have been irrevocably delivered to the human brain to treat Parkinson's be controlled if the genes unexpectedly start causing problems?
The researchers also said prior Parkinson's experiments using growth factors -- naturally occurring substances that cause cells to grow and divide -- to rescue dying brain cells may have failed because they occurred too late in the course of the disease.
We have worked every day for 10 years to design a construct to the gene delivery vector that enhances the safety profile of gene transfer for Parkinson's disease,
Mandel said. With that added measure of safety, we believe we can intervene with gene transfer in patients at earlier stages of the disease. We strongly believe that trials to save dopamine-producing connections in patients with Parkinson's disease have failed because the therapy went into patients who were in the late stages of the disease and who had too few remaining dopamine-producing connections.
The study appears in the September issue of the journal Molecular Therapy.
Source: United Press International
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