CAG Repeat Expansion Diseases: From Basic Biology to Therapeutics

Trinucleotide CAG expansion mutations cause a number of progressive neurodegenerative disorders including Huntington’s disease (HD), the spinocerebellar ataxias (SCAs) 1, 2, 3, 6, 7 and 17, and spinal and bulbar muscular atrophy (SBMA). The pathogenesis caused by protein or RNA gain-of-function effects at these loci is being investigated at multiple levels, giving us a new appreciation for the complexity of changes imparted by these mutations, and opening the door for novel therapeutic approaches. The 2015 Gordon Conference on CAG Triplet Repeat Disorders and the associated Gordon Research Seminar (GRS) will bring together scientists from around the world to discuss cutting-edge research on the molecular and cellular mechanisms of these disorders and the state-of-the-art therapeutic strategies being developed to combat them. Meeting topics will include: RNA as a key to pathogenesis; advances in imaging; new animal models; designing therapies; emerging drug targets; mechanisms of pathogenesis, and systemic disease. Strong emphasis will be placed on training and mentoring of young scientists with time devoted to career issues. All participants will be required to present posters and some poster presenters will be selected for short talks. Additionally, top posters and oral presentations from the GRS will take part in a focused GRC session. The collegial atmosphere of this Conference, with structured discussion sessions and opportunities for informal afternoon and evening gatherings will provide an excellent opportunity for scientists from different disciplines to brainstorm with each other and to establish interdisciplinary collaborations.