Abstract The 2019 Gordon Research Conference on CAG Triplet Repeat Disorders will gather leading scientists from around the world to discuss novel high-impact research on CAG Triplet Repeat Disorders. The family of CAG triplet repeat diseases includes Huntington’s disease (HD), the spinocerebellar ataxias (SCAs) 1, 2, 3, 6, 7, 12 and 17, spinal and bulbar muscular atrophy (SBMA) and dentatorubral pallidoluysian atrophy (DRPLA). The objective of the conference is to promote interdisciplinary exchange of information and approaches. The conference will bring together leading senior and rising junior researchers, with a broad range of experts from related areas with high relevance to the investigation of CAG repeat diseases, and foster diversity of speakers and discussion leaders. While this is the tenth conference in this very successful series, it is worth noting what is new and different at this stage of research into CAG triplet repeat disorders. We will have a session (the first for the conference series) on disease-modifying clinical trials. We will consider exciting new developments in disease biomarkers. We will focus on the provocative new findings that CAG repeat expansion disease genetic modifiers increasingly appear to be related to DNA repair, revealing new vistas for therapeutic targets validated by human clinical data. We will have the opportunity to consider the first three-dimensional structures of polyglutamine containing proteins, and incorporate those insights into understanding the role of post-translational modifications, also revealing many novel potential therapeutic targets. There will be a GRS, which will include a mentorship component, and bring together senior and early career scientists together. Understanding the molecular foundations of CAG repeat diseases will enable the development of new diagnostic and therapeutic strategies. Translation will be encouraged by the combination of basic scientists, translational researchers and clinical researchers, including clinical trials. In summary, we have developed an exciting scientific program (as described in the application) that emphasizes emerging themes in pathogenic mechanisms of the CAG repeat disorders, as well as novel diagnostic techniques, and potentially disease- modifying therapeutic approaches.

Narrative The 2019 Gordon Research Conference on CAG Triplet Repeat Disorders will gather leading scientists from around the world to discuss novel high-impact research on CAG Triplet Repeat Disorders. We have developed an exciting scientific program as described in the application that emphasizes emerging themes in pathogenic mechanisms of the CAG repeat disorders, as well as novel diagnostic techniques, and potentially disease- modifying therapeutic approaches.