Common mutation successfully targeted in Lou Gehrig's disease and frontotemporal dementia

August 14, 2014 4:04 PM

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Common mutation successfully targeted in Lou Gehrig's disease and frontotemporal dementia

JUPITER, FL, August 14, 2014 – An international team led by scientists from the Florida campuses of The Scripps Research Institute (TSRI) and the Mayo Clinic have for the first time successfully designed a therapeutic strategy targeting a specific genetic mutation that causes a common form of amyotrophic lateral sclerosis (ALS), better known as Lou Gehrig's disease, as well a type of frontotemporal dementia (FTD).

The scientists developed small-molecule drug candidates and showed they interfere with the synthesis of an abnormal protein that plays a key role in causing both diseases. The team also developed biomarkers that can test the efficacy of this and other therapies.

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