Sunday
2:00 pm - 9:00 pm
Arrival and Check-in
6:00 pm - 7:00 pm
Dinner
7:30 pm - 7:40 pm
Introductory Comments by GRC Staff / Welcome and Introduction from the Chairs
7:40 pm - 9:30 pm
Keynote Session: Molecular Mechanisms and Biomarkers in Neurodevelopmental Disorders
Discussion Leader: Peter Kind (University of Edinburgh, United Kingdom)
7:40 pm - 7:45 pm
Introduction by Discussion Leader
7:45 pm - 8:15 pm
Craig Erickson (Cincinnati Childrens Hospital, United States)
"Individual Variation as the Foundation for Personalized Medicine in Fragile X Syndrome"
8:15 pm - 8:20 pm
Discussion
8:20 pm - 8:50 pm
Emily Osterweil (Boston Children's Hospital/Harvard Medical School, United States)
"Dysregulated Translation in Fragile X: The Long and Short of it"
8:50 pm - 8:55 pm
Discussion
8:55 pm - 9:25 pm
Michael Gandal (University of Pennsylvania, United States)
"Interpreting Genetic Risk for ASD Through the Lens of Neurodevelopment"
9:25 pm - 9:30 pm
Discussion
Monday
7:30 am - 8:30 am
Breakfast
9:00 am - 12:30 pm
Molecular and Cellular mechanisms of Fragile X Syndrome and Related Disorders
Discussion Leader: Emily Osterweil (Boston Children's Hospital/Harvard Medical School, United States)
9:00 am - 9:25 am
Joel Richter (University of Massachusetts Medical School, United States)
"Splicing Dysregulation in Fragile X Syndrome"
9:25 am - 9:35 am
Discussion
9:35 am - 10:00 am
Peter Todd (University of Michigan, United States)
"CGG Repeats as Functional Elements and Drivers of Disease"
10:00 am - 10:10 am
Discussion
10:10 am - 10:40 am
Coffee Break
10:40 am - 11:05 am
Debby Silver (Duke University Medical Center, United States)
"Developmental and Molecular Mechanisms Underlying DDX3X Syndrome"
11:05 am - 11:15 am
Discussion
11:15 am - 11:40 am
Late-Breaking Topic
11:40 am - 11:50 am
Discussion
11:50 am - 12:00 pm
Short Talk Selected from Poster Abstracts
12:00 pm - 12:05 pm
Discussion
12:05 pm - 12:15 pm
Short Talk Selected from Poster Abstracts
12:15 pm - 12:20 pm
Discussion
12:20 pm - 12:30 pm
Poster Previews
12:30 pm - 1:30 pm
Lunch
1:30 pm - 4:30 pm
Free Time
3:00 pm - 4:00 pm
The GRC Power Hourâ„¢
The GRC Power Hourâ„¢ is designed to address diversity and inclusion in the scientific workplace by providing a safe environment for informal and meaningful conversations amongst colleagues of all career stages. The program supports the professional growth of all members of our communities, including ethnicity, race and/or gender identity by providing an open forum for discussion and mentoring.
Organizer: Liz McCullagh (Oklahoma State University, United States)
4:30 pm - 6:00 pm
Poster Session
6:00 pm - 8:00 pm
Advances in Genetics and Functional Genomics of ASDs
Discussion Leader: Michael Gandal (University of Pennsylvania, United States)
6:00 pm - 6:25 pm
Varun Warrier (University of Cambridge, United Kingdom)
"What is Autism? Using Genetics to Understand Heterogeneity in Autism"
6:25 pm - 6:35 pm
Discussion
6:35 pm - 7:00 pm
Hyejung Won (University of North Carolina at Chapel Hill, United States)
"Massively Parallel Reporter Assays Identify Shared Neurobiology Among Psychiatric Disorders"
7:00 pm - 7:10 pm
Discussion
7:10 pm - 7:35 pm
Brett Trost (The Hospital for Sick Children, Canada)
"The Genomic Architecture of Autism: From Single Nucleotide Variants to Tandem Repeat Expansions"
7:35 pm - 7:45 pm
Discussion
7:45 pm - 7:55 pm
Short Talk Selected from Poster Abstracts
7:55 pm - 8:00 pm
Discussion
8:00 pm - 9:00 pm
Dinner
Tuesday
7:30 am - 8:30 am
Breakfast
8:30 am - 9:00 am
Group Photo
9:00 am - 12:30 pm
Insights from Human Cellular Models of Fragile X and ASD
Discussion Leader: Sebastian Jacquemont (University of Montréal, Sainte Justine Hospital, Canada)
9:00 am - 9:25 am
Alysson Muotri (UC San Diego, United States)
"Modeling and Treatment of ASD Using Human Brain Organoids"
9:25 am - 9:35 am
Discussion
9:35 am - 10:00 am
Alex Shcheglovitov (University of Utah, United States)
"Exploring the Impact of Rare Autism-Linked Mutations on Human Telencephalic Development Using Stem Cell-Derived Organoids"
10:00 am - 10:10 am
Discussion
10:10 am - 10:40 am
Coffee Break
10:40 am - 11:05 am
Lindy Barrett (Broad Institute, United States)
"Leveraging iPSC Models to Study Neural Development and Disease"
11:05 am - 11:15 am
Discussion
11:15 am - 11:40 am
Mustafa Sahin (Boston Children's Hospital, United States)
"Modeling TSC in Stem Cell Derived Neurons"
11:40 am - 11:50 am
Discussion
11:50 am - 12:00 pm
Short Talk Selected from Poster Abstracts
12:00 pm - 12:05 pm
Discussion
12:05 pm - 12:15 pm
Short Talk Selected from Poster Abstracts
12:15 pm - 12:20 pm
Discussion
12:20 pm - 12:30 pm
Poster Previews
12:30 pm - 1:30 pm
Lunch
1:30 pm - 4:30 pm
Free Time
4:30 pm - 6:00 pm
Poster Session
6:00 pm - 8:00 pm
Cellular Heterogeneity in Fragile X and ASD
Discussion Leader: Eric Klann (New York University, United States)
6:00 pm - 6:25 pm
Robert Darnell (Rockefeller University, United States)
"FMRP RNA Regulation Within Brain Neurons"
6:25 pm - 6:35 pm
Discussion
6:35 pm - 7:00 pm
Laura Andreae (King's College London, United Kingdom)
"Cell-Type Specific Synaptic Alterations in Mouse Models for ASD"
7:00 pm - 7:10 pm
Discussion
7:10 pm - 7:35 pm
Late-Breaking Topic
7:35 pm - 7:45 pm
Discussion
7:45 pm - 7:55 pm
Short Talk Selected from Poster Abstracts
7:55 pm - 8:00 pm
Discussion
8:00 pm - 9:00 pm
Dinner
Wednesday
7:30 am - 8:30 am
Breakfast
9:00 am - 12:30 pm
Sensory/Cognitive Dysfunction and Biomarkers in Fragile X and ASD
Discussion Leader: Michela Fagiolini (Boston Children's Hospital, United States)
9:00 am - 9:25 am
Ernest Pedapati (Cincinnati Children's Research Foundation / University of Cincinnati, United States)
"Constraints and Excesses: Lessons from Human Fragile X Brain Physiology"
9:25 am - 9:35 am
Discussion
9:35 am - 10:00 am
Lauren Schmitt (Cincinnati Children's Hospital Medical Center, United States)
"A Paradigm Shifting View of Fragile X Syndrome: Leveraging Molecular Biomarkers to Rethink Intellectual Disability in this Single Gene Disorder"
10:00 am - 10:10 am
Discussion
10:10 am - 10:40 am
Coffee Break
10:40 am - 11:05 am
Carlos Portera-Cailliau (David Geffen School of Medicine at UCLA, United States)
"Interneurons, Circuits, and Behavior in Fragile X Syndrome"
11:05 am - 11:15 am
Discussion
11:15 am - 11:40 am
Jason Lerch (University of Oxford, United Kingdom)
"Identifying Molecular Subtypes in Idiopathic Autism and Related Neurodevelopmental Disorders"
11:40 am - 11:50 am
Discussion
11:50 am - 12:00 pm
Short Talk Selected from Poster Abstracts
12:00 pm - 12:05 pm
Discussion
12:05 pm - 12:15 pm
Short Talk Selected from Poster Abstracts
12:15 pm - 12:20 pm
Discussion
12:20 pm - 12:30 pm
Poster Previews
12:30 pm - 1:30 pm
Lunch
1:30 pm - 4:30 pm
Free Time
4:30 pm - 6:00 pm
Poster Session
6:00 pm - 8:00 pm
Synapse and Circuit Mechanisms of FXS and ASD
Discussion Leader: Helen Bateup (University of California, Berkeley, United States)
6:00 pm - 6:25 pm
Claudia Bagni (University of Lausanne, Switzerland)
"Alterations in Structural and Functional Plasticity in the Striatum in Neurodevelopmental Disorders"
6:25 pm - 6:35 pm
Discussion
6:35 pm - 7:00 pm
Pablo Castillo (Albert Einstein College of Medicine, United States)
"FMRP and Local Protein Synthesis at the Synapse"
7:00 pm - 7:10 pm
Discussion
7:10 pm - 7:35 pm
Late-Breaking Topic
7:35 pm - 7:45 pm
Discussion
7:45 pm - 7:55 pm
Short Talk Selected from Poster Abstracts
7:55 pm - 8:00 pm
Discussion
8:00 pm - 9:00 pm
Dinner
Thursday
7:30 am - 8:30 am
Breakfast
8:30 am - 9:00 am
Business Meeting
Nominations for the Next Vice Chair(s); Complete the GRC Evaluation Forms; Discuss Future Dates and Venue; Election of the Next Vice Chair(s)
9:00 am - 12:30 pm
Advances in Therapeutics and Clinical Trial Design
Discussion Leader: Michael Tranfaglia (FRAXA Research Foundation, United States)
9:00 am - 9:25 am
Steven Gray (University of Texas Southwestern Medical Center, United States)
"Gene Therapy for Fragile X Syndrome and Other Neurodevelopmental Disorders"
9:25 am - 9:35 am
Discussion
9:35 am - 10:00 am
Elizabeth Berry Kravis (Rush University Medical Center, United States)
"Development of Phosphodiesterase Inhibitors to Target Underlying Neural Mechanisms and Cognition in Fragile X Syndrome"
10:00 am - 10:10 am
Discussion
10:10 am - 10:40 am
Coffee Break
10:40 am - 11:05 am
Paul Wang (Simons Foundation / CRA, United States)
"Trials (and Tribulations) of Arbaclofen: Testing E:I Biomarkers, Refining Clinical Trial Design, Exploring New Endpoints, and Thinking About Efficacy"
11:05 am - 11:15 am
Discussion
11:15 am - 11:40 am
Kemi Olugemo (Ultragenyx, United States)
"Methodological Considerations for Clinical Trials in Angelman Syndrome"
11:40 am - 11:50 am
Discussion
11:50 am - 12:00 pm
Short Talk Selected from Poster Abstracts
12:00 pm - 12:05 pm
Discussion
12:05 pm - 12:15 pm
Short Talk Selected from Poster Abstracts
12:15 pm - 12:20 pm
Discussion
12:20 pm - 12:30 pm
Poster Previews
12:30 pm - 1:30 pm
Lunch
1:30 pm - 4:30 pm
Free Time
4:30 pm - 6:00 pm
Poster Session
6:00 pm - 8:00 pm
Body-Brain Interactions in ASD
Discussion Leader: Ellen Hoffman (Yale University, United States)
6:00 pm - 6:25 pm
Gaia Novarino (IST Austria, Austria)
"Defining the Phenotypic Landscape of Autism Spectrum Disorders at Single-Cell Resolution"
6:25 pm - 6:35 pm
Discussion
6:35 pm - 7:00 pm
Gloria Choi (Massachusetts Institute of Technology, United States)
"Neuroimmune Interactions Shaping Social Behavior"
7:00 pm - 7:10 pm
Discussion
7:10 pm - 7:35 pm
Late-Breaking Topic
7:35 pm - 7:45 pm
Discussion
7:45 pm - 7:55 pm
Short Talk Selected from Poster Abstracts
7:55 pm - 8:00 pm
Discussion
8:00 pm - 9:00 pm
Dinner
Friday
7:30 am - 8:30 am
Breakfast
9:00 am
Departure