This application requests funding for the 2015 Gordon Research Conference on CAG Triplet Repeat Disorders and the associated Graduate Research Seminar to be held at the Il Ciocco Resort in Barga, Italy from May 30 - June 5, 2015. This will be the eighth Gordon Research Conference on CAG Triplet Repeat Disorders. The previous five conferences have alternated between American (Mount Holyoke College, 2001, 2005; Waterville Valley NH, 2009 and 2013) and European (Il Ciocco, Italy 2003, 2011 and Aussois, France 2007) sites. This is the third year that there will be an associated Graduate Research Seminar. The CAG Triplet Repeat Disorders are a group of largely untreatable inherited neurological disorders which result from an expansion in a CAG trinucleotide repeat in the mutant genes. This group of diseases includes Huntington's disease (HD), spinal and bulbar muscular atrophy (SBMA, Kennedy's disease), spinocerebellar ataxias types 1, 2, 3, 6, 7, and 17, and dentatorubropallidoluysian atrophy (DRPLA). The disease-inducing CAG repeat expansion occurs in coding regions within the affected gene and can confer toxicity via RNA-dependent and/or protein-dependent mechanisms. Differences in the anatomical distribution of selective neuronal degeneration make it imperative to unravel their natural histories, cellular dysfunction and gene, RNA and protein pathways involved in the pathogenesis. To increase the pace of basic research discovery and set in place the contacts and clinical resources necessary to move the basic science forward, perhaps into the clinic, a multidisciplinary research effort is required. It is essential that collaborative projects between scientists from diverse disciplines ranging from organic chemistry and fruit fly genetics to neurology and human clinical trials be established. The conference on CAG Triplet Repeat Disorders will gather together young investigators and established senior scientists to deliver provoking lectures on the cutting-edge of science. In keeping with the Gordon Research Conference format, there will be generous time allocated for both structured discussions led by peers and for informal discussion and social interactions to facilitate collaboration. Strong emphasis is placed on training and mentoring of young scientists, and time will be devoted to career issues. All participants will be required to present posters. The 2015 GRC has new innovations including a devoted session within the GRC to GRS participants and more late-breaking talks to be pulled from the abstracts from prior years. Priority will be given to women, minorities and persons with disabilities when selecting participants for all sessions.
The 2015 Gordon Research Conference on CAG Triplet Repeat Disorders and its associated Graduate Research Seminar will bring together researchers and clinicians to discuss cutting-edge information on disease mechanisms and therapeutic interventions for these devastating neurological diseases. In addition, the format of the Gordon Research Conference and the funding sought herein will promote and ensure the attendance and enhanced education of junior scientists, including graduate students, postdoctoral fellows, and junior faculty.