Cornell University Archives

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.

Cornell University Researcher Looks to Restore Fragile X Protein in Neurons

July 8, 2016

Which is the right FMRP for therapeutic development of Fragile X syndrome? When researchers develop effective drugs that reactivate FMRP — the protein that is normally silenced in Fragile X — what in the world will they do next? So ponders Cornell University researcher Samie R. Jaffrey, MD, PhD. Jaffrey, professor, Pharmacology, Weill Cornell Medical College, Cornell University, knows reactivating FMRP will lead to many important questions, such as: Which cell type needs FMRP? How much FMRP protein is needed to restore brain function? Where in the brain will FMRP protein be needed? Where in a neuron will the FMRP needs to be expressed?

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.

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.