Alan Kozikowski PhD, University of Illinois, Chicago
Synthesis and preliminary (ADME) testing of selective HDAC inhibitors for treatment of a mouse model of Rett Syndrome
Histone deacetylase (HDAC) enzymes are proteins that regulate gene expression and are involved in brain cell degeneration in neurodegenerative diseases. HDAC inhibitors are drugs which block the action of HDAC enzymes and have shown promise as potential treatments for neurodegenerative diseases such as Parkinson's, Motor Neuron and Huntington's diseases. These drugs have also recently been suggested as potential treatments for neurodevelopmental disorders such as Fragile X, RubinsteinTaybi syndrome and Rett syndrome. Dr. Kozikowski was awarded a short-term contract to fund preliminary predictive tests to select the best candidate drug from a lead series for testing in a Rett mouse model.
IRSF conducts thorough due diligence on all research grants and for clinical studies involving patients with Rett syndrome, since safety is of paramount importance. We accomplish this through a rigorous peer-review process, drawing on the external advice and expertise of leading experts and specialists in the fields of Rett syndrome, neuroscience, autism, drug discovery and development and other relevant disciplines as appropriate.
IRSF will shortly announce additional detail on the two new funding mechanisms and will solicit research programs through an online grants submission process. Dr. Horton commented "With the launch of these new award mechanisms, we will have greater power and flexibility to accelerate drug discovery and to develop and test new treatments. These awards will bring us another step closer to finding a cure through the pharmacological reversal of Rett syndrome."
According to Stephen Bajardi, Executive Director of IRSF, "It is through the combined efforts of our parent volunteers, generous con
|Contact: Dr. Antony Horton|
International Rett Syndrome Foundation