Samie Jaffrey, PhD, at Weill Medical College of Cornell University, FRAXA research grant

Which is the right FMRP for Therapeutic Development of Fragile X Syndrome?

April 24, 2017

Many forms of FMRP exist in the brain. This project aims to pinpoint which versions of the protein are most critical to restore for effective Fragile X treatments.

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Molecular Mechanisms of Cytoskeletal Regulation by FMRP

January 9, 2015

With FRAXA funding, Dr. Jaffrey linked FMR1 loss to abnormal dendritic spines via RhoA signaling, suggesting RhoA-targeted therapies could help treat Fragile X.

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Samie Jaffrey, PhD, at Weill Medical College of Cornell University, FRAXA research grant

Scientists Uncover Trigger for Fragile X Syndrome

February 27, 2014

A new study led by Weill Cornell Medical College scientists shows that Fragile X syndrome occurs because of a mechanism that shuts off the gene associated with the disease. The findings, published today in Science, also show that a compound that blocks this silencing mechanism can prevent Fragile X syndrome – suggesting a similar therapy may be possible for 20 other diseases that range from mental retardation to multisystem failure.

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FRAXA Funded Research

Current Research Grants (38)