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Miracle Molecule

Scripps Research Institute in Jupiter - Mathew Disney - fragile X syndrome   Scientists at Scripps Research Institute in Jupiter have designed a compound that reverses some of the complications with fragile X syndrome, the only known single-gene cause of autism. The condition, which can lead to mental retardation, memory impairment and autism, is closely linked to tremor ataxia syndrome, which normally affects men older than 50 and results in Parkinson’s-like symptoms. Both fragile X and tremor ataxia syndrome are caused by a structural motif known as expanded triplet repeat, in which a series of three nucleotides are repeated more than normal, causing problems with RNA processing. While the compound is still in the early stages of testing, it is capable of targeting the right RNA and reversing the defects that cause tremor ataxia, according to Mathew Disney, the Scripps researcher who led the study.
 

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