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Compounds for Suppressing Fragile X Premutation rCGG Repreat-Mediated Toxicity
Application Compounds for the treatment of Fragile X-associated tremor/ataxia syndrome. Key Benefits 11 known, FDA approved drugs identified that reduced rCGG-mediated lethality to a certain degree. 3 of these drugs also rescued locomotion deficits. Technical Summary Fragile X syndrome (FXS) is the most common inheritable form of cognitive disabi...
Published: 2/24/2011       Inventor(s): Peng Jin, Abrar Qurashi
Compounds for the Treatment of Fragile X Syndrome and Methods of Screening
Application Small molecule compounds that may have therapeutic efficacy in Fragile X Syndrome and chemical genetic screens for potential drug targets. Technical Summary Fragile X Syndrome (FXS), the most common heritable form of mental retardation, is caused by transcriptional misregulation of the FMR1 gene and a subsequent loss of function of t...
Published: 2/24/2009       Inventor(s): Stephen Warren, Shuang Chang, Peng Jin