Atsugi
Moderator emeritus
- Joined
- Jan 11, 2011
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- Lost a loved one
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- 12/2010
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Does this mean that they’ve developed a drug that can prevent C9orf72-type ALS?
Extracted from a Mayo Clinic Press Release, August 14th, 2014:
JACKSONVILLE, Fla. — A team of researchers at Mayo Clinic and The Scripps Research Institute in Florida have developed a new therapeutic strategy to combat the most common genetic risk factor for the neurodegenerative disorders amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease) and frontotemporal dementia (FTD). In the Aug. 14 issue of Neuron, they also report discovery of a potential biomarker to track disease progression and the efficacy of therapies.
The scientists developed a small-molecule drug compound to prevent abnormal cellular processes caused by a mutation in the C9ORF72gene. The findings come on the heels of previous discoveries by Mayo investigators that the C9ORF72 mutation produces an unusual repetitive genetic sequence that causes the buildup of abnormal RNA in brain cells and spinal cord.
Extracted from a Mayo Clinic Press Release, August 14th, 2014:
JACKSONVILLE, Fla. — A team of researchers at Mayo Clinic and The Scripps Research Institute in Florida have developed a new therapeutic strategy to combat the most common genetic risk factor for the neurodegenerative disorders amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease) and frontotemporal dementia (FTD). In the Aug. 14 issue of Neuron, they also report discovery of a potential biomarker to track disease progression and the efficacy of therapies.
The scientists developed a small-molecule drug compound to prevent abnormal cellular processes caused by a mutation in the C9ORF72gene. The findings come on the heels of previous discoveries by Mayo investigators that the C9ORF72 mutation produces an unusual repetitive genetic sequence that causes the buildup of abnormal RNA in brain cells and spinal cord.