What unique areas of research into Rett syndrome and other neurological disorders are being pursued?
A. Current efforts are ongoing along both clinical and basic research pathways. We believe that one day these will converge as treatment trials come online addressing a fundamental cure. From the clinical perspective, through our Rare Disease grant, we are examining the natural history and phenotype-genotype correlations of RTT in order to be in good position for clinical trials. At the basic level, research in the Pozzo-Miller and Sweatt laboratories is examining the basic role of the RTT gene in brain function, and a new initiative in collaboration with the Pozzo-Miller, Townes and Strong laboratories, along with major support from core grant noted above, is pursuing the development of pluripotent stem cells from skin fibroblasts expressing specific RTT mutations as another vehicle to evaluate the affect of these mutations on nerve-cell development and interaction and to be used as testbeds for evaluating specific therapies. Q. Have there been any recent breakthroughs in the treatmen
