FRAXA Grant to Alan Tartakoff, PhD — Case Western Reserve University

FRAXA Awards: $63,000 in 2000 $30,000 in 1999 $30,000 in 1998   Transport of the Fragile X Protein: Generation of monoclonal antibodies to FMRP, FXR1 and FXR2 by Alan Tartakoff, 7/1/2000 My cell biology research laboratory is focused on three aspects of the biology of FMRP, the protein which is missing in the Fragile X Syndrome. 1. Independent of the functions of FMRP, we have taken the first steps toward developing a protein-therapeutic strategy for the Fragile X Syndrome. This intiative stems from recent success in delivering other proteins into the cytoplasm and nucleus of cells, both when working with cells in culture and in experiments with mice. For example, when appropriately modified forms of proteins are injected into the peritoneal cavity of mice, they arrive – within hours – inside cells throughout the body, including many nerve cells of the brain. In order for such delivery to occur, the

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