Conference Description
ATTENTION: The GRC bus picks up and drops off at the Florence Airport (FLR) only. GRC no longer has bus service to / from the Pisa Airport.
The Fragile X and Autism-Related Disorders GRC is a premier, international scientific conference focused on advancing the frontiers of science through the presentation of cutting-edge and unpublished research, prioritizing time for discussion after each talk and fostering informal interactions among scientists of all career stages. The conference program includes a diverse range of speakers and discussion leaders from institutions and organizations worldwide, concentrating on the latest developments in the field. The conference is five days long and held in a remote location to increase the sense of camaraderie and create scientific communities, with lasting collaborations and friendships. In addition to premier talks, the conference has designated time for poster sessions from individuals of all career stages, and afternoon free time and communal meals allow for informal networking opportunities with leaders in the field.
Genetic syndromes with increased prevalence of autism spectrum disorder (ASD) and intellectual disability (ID) offer an opportunity to understand the brain pathophysiology that manifests as ASD; this knowledge can suggest potential targeted therapies. It is now clear that the genetics underlying ASD are complex; with at least several hundred genes conferring small amounts of risk. Studies utilizing genomic sequencing and found several rare variants in individuals with ASD. These studies suggest a high degree of convergence on particular cellular processes and biochemical pathways, suggesting there may be convergence of underlying mechanisms and potential therapeutic strategies. The study of monogenic or syndromic forms of ASD, has been a leading strategy to gain insight into the complex mechanisms of ASD. Fragile X Syndrome (FXS) is one of the most common inherited forms of ID and the leading single gene cause of ASD. Since the Fragile X gene (FMR1) was cloned in 1991, the field has used cellular assays and model organisms to elucidate the functions of the FMR1 protein (FMRP), the consequences of its loss and identify therapeutic targets for FXS and ASD. Other syndromic forms of ASD, such as tuberous sclerosis complex, Rett Syndrome, Angelman Syndrome and others, are being investigated using similar approaches. Recent technological advances in human stem-cell derived neurons, organoids, single cell sequencing, gene therapy and novel model organisms are setting the stage for transformative advances in therapeutic development for these neurodevelopmental disorders. This conference will bring together leading scientists and clinicians studying FXS, ASD and related neurodevelopmental disorders with the ultimate goal of identifying the mechanisms that underlie the heterogeneous symptomatology associated with these disorders and the best potential pathways for translating this knowledge into clinical trials.
The topics, speakers, and discussion leaders for the conference sessions are displayed below. The conference chair is currently developing their detailed program, which will include the complete meeting schedule, as well as the talk titles for all speakers. The detailed program will be available by January 13, 2024. Please check back for updates.
Keynote Session: Molecular Mechanisms and Biomarkers in Neurodevelopmental Disorders
Discussion Leaders
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Peter Kind (University of Edinburgh, United Kingdom)
Speakers
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Emily Osterweil (University of Edinburgh, United Kingdom)
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Craig Erickson (Cincinnati Childrens Hosp, United States)
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Michael Gandal (University of Pennsylvania, United States)
Molecular and Cellular mechanisms of Fragile X Syndrome and Related Disorders
Discussion Leaders
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Emily Osterweil (University of Edinburgh, United Kingdom)
Speakers
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Joel Richter (University of Massachusetts Medical School, United States)
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Debby Silver (Duke University Medical Center, United States)
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Peter Todd (University of Michigan, United States)
Advances in Genetics and Functional Genomics of ASDs
Discussion Leaders
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Michael Gandal (University of Pennsylvania, United States)
Speakers
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Hyejung Won (UNC, United States)
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Brett Trost (The Hospital for Sick Children, Canada)
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Varun Warrier (University of Cambridge, United Kingdom)
Insights from Human Cellular Models of Fragile X and ASD
Discussion Leaders
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Sebastian Jacquemont (University of Montréal, Sainte Justine Hospital, Canada)
Speakers
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Alysson Muotri (UC San Diego, United States)
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Alex Shcheglovitov (University of Utah, United States)
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Lindy Barrett (Broad Institute, United States)
Cellular Heterogeneity in Fragile X and ASD
Discussion Leaders
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Eric Klann (New York University, United States)
Speakers
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Robert Darnell (Rockefeller University, United States)
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Oscar Marin (King's College London, United Kingdom)
Sensory/Cognitive Dysfunction and Biomarkers in Fragile X and ASD
Discussion Leaders
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David Nelson (Baylor College of Medicine, United States)
Speakers
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Ernest Pedapati (Cincinnati Children's Research Foundation / University of Cincinnati, United States)
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Carlos Portera-Cailliau (David Geffen School of Medicine at UCLA, United States)
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Lauren Schmitt (Cincinnati Children's Hospital Medical Center, United States)
Synapse and Circuit Mechanisms of FXS and ASD
Discussion Leaders
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Helen Bateup (University of California, Berkeley, United States)
Speakers
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Jason Lerch (University of Oxford, United Kingdom)
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Claudia Bagni (University of Lausanne, Switzerland)
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Pablo Castillo (Albert Einstein College of Medicine, United States)
Advances in Therapeutics and Clinical Trial Design
Discussion Leaders
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Michael Tranfaglia (FRAXA Research Foundation, United States)
Speakers
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Steven Gray (University of Texas Southwestern Medical Center, United States)
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Elizabeth Berry Kravis (Rush University Medical Center, United States)
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Scott Stromatt (Ultragenyx, United States)
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Paul Wang (Simons Foundation / CRA, United States)
Body-Brain Interactions in ASD
Discussion Leaders
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Ellen Hoffman (Yale University, United States)
Speakers
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Gaia Novarino (IST Austria, Austria)
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Gloria Choi (Massachusetts Institute of Technology, United States)
The GRC Power Hourâ„¢
Organizers
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Liz McCullagh (Oklahoma State University, United States)