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First Steps
We are off and running! Topping the list of CDI strategies is the pursuit of “low-hanging fruit”: approved drugs or compounds that have been developed for other diseases. Tens of thousands of compounds are already in existence, and like levodopa that was used experimentally on some patients with great success (for what turned out to be a form of dopa-responsive dystonia), treatments and cures may already be out there and simply awaiting testing. The first of these clinical trials are underway.
In addition to pursuing existing drugs, CDI is also funding research for novel therapeutics. One of the projects focuses on the use of RNA interference (RNAi) as a means to silence the mutant protein for DYT1 dystonia. This is an enormously important avenue of research since DYT1 appears to be a particularly attractive target for RNAi therapy. A collaborative study to test selected siRNAs is also up and running. These exciting first steps have been made possible by the generous support of individuals who endorse our mission and our vision. |
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