Common Themes in Diverse Diseases: From Molecular Mechanisms to Therapeutic Strategies

This meeting is over-subscribed. Applications received as of 4/20/23 will be considered after the oversubscribed deadline of 5/7/23 if spots are available.

The CAG triplet repeat disorders include 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). They are all rare and all currently untreatable. However, these are exciting and promising times in the field, with several therapeutics currently being tested in the clinic. Treatments in development include multiple modalities from small molecules to advanced gene-based therapeutics.

The ongoing identification of viable disease-modifying therapies is critically dependent on basic research that defines the fundamental mechanisms underlying pathogenesis. New insights are uncovering mechanisms that are common among diseases as well as those that may underpin specific aspects of each disease. Common mechanisms also extend beyond CAG repeat expansion disorders to those caused by expansions in other types of repeats. The multiple-disease nature of this conference allows cross-fertilization of research from one disease to another, enhancing our understanding of both underlying biology and therapeutic opportunities.

At the 2023 Gordon Research Conference on CAG Triplet Repeat Disorders we will hear from researchers at the cutting edge of defining basic disease mechanisms and those working on clinical translation and therapeutic trials. The conference will explore the wide variety of pathobiological dysfunction that can occur at the level of DNA, RNA or protein species, mechanistic intersections among repeat expansion diseases, and emerging technologies that open doors to new repeat disease discovery, diagnosis and enhanced resolution of underlying pathogenic pathways. The conference will bring together leading senior and rising junior researchers, with a broad range of expertise, including neurobiologists, pathologists, geneticists, biochemists, cellular and molecular biologists, structural biologists, computer scientists, clinicians and clinical researchers, and foster diversity of speakers and discussion leaders. There will be ample time for critical discussion, and opportunities for young investigators to present on emerging new data by including talks selected from submitted poster abstracts, integrated into all sessions.