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: Genetic Landscape of Neurodegenerative Disorders
Discussion Leader: Philip De Jager (Columbia University Medical Center, United States)
7:40 pm - 7:50 pm
Introduction by Discussion Leader
7:50 pm - 8:50 pm
Alison Goate (Icahn School of Medicine at Mount Sinai, United States)
"The Genetic Landscape of Alzheimer's Disease and How This Informs Disease Mechanism"
8:50 pm - 9:20 pm
Discussion
9:20 pm - 9:30 pm
General Discussion
Monday
7:30 am - 8:30 am
Breakfast
9:00 am - 12:30 pm
Recent Advances in Genetics and Genomic Approaches to Neurodegenerative Disease
Discussion Leader: Alison Goate (Icahn School of Medicine at Mount Sinai, United States)
9:00 am - 9:05 am
Introduction by Discussion Leader
9:05 am - 9:25 am
Philip De Jager (Columbia University Medical Center, United States)
"Deconstructing the Cellular Communities that Drive Trajectories to Alzheimer's Disease and Alternative Brain Aging"
9:25 am - 9:35 am
Discussion
9:35 am - 9:55 am
Li-Huei Tsai (Picower Institute for Learning and Memory, Massachusetts Institute of Technology, United States)
"Single Cell Dissection of Dementia Pathogenesis"
9:55 am - 10:05 am
Discussion
10:05 am - 10:15 am
Jack Humphrey (Icahn School of Medicine at Mount Sinai, United States)
"Integrative Functional Genomics in Post-Mortem Tissues from the ALS/FTD Disease Spectrum Identify New Biomarkers and Mechanisms for Patient Stratification"
10:15 am - 10:20 am
Discussion
10:20 am - 10:30 am
Steven Boggess (University of California, San Francisco, United States)
"Uncovering Mechanisms of Hyperexcitability in Neurodegenerative Disease with Functional Genomics"
10:30 am - 10:35 am
Discussion
10:35 am - 11:00 am
Coffee Break
11:00 am - 11:20 am
Mark Cookson (National Institute on Aging, NIH, United States)
"Regulation of LRRK2 Expression Links Parkinson's to Inflammation"
11:20 am - 11:30 am
Discussion
11:30 am - 11:50 am
Sarah Marzi (King's College London, United Kingdom)
"Epigenetic Signatures of Genetic Risk in Alzheimer's Disease"
11:50 am - 12:00 pm
Discussion
12:00 pm - 12:10 pm
Julia Obergasteiger (Cervo brain research center, Canada)
"Function and Neuroprotective Potential of Flcn Knockout in Parkinson’s Disease"
12:10 pm - 12:15 pm
Discussion
12:15 pm - 12:25 pm
Jean-Pierre Roussarie (Boston University School of Medicine, United States)
"Regulation of Entorhinal Cortex Vulnerability by TENM3, a Genetic Susceptibility Factor for Alzheimer’s Disease"
12:25 pm - 12:30 pm
Discussion
12:30 pm - 1:30 pm
Lunch
1:30 pm - 4:30 pm
Free Time
3:00 pm - 4:00 pm
The GRC Power Hour™
The GRC Power Hour™ 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: Hemali Phatnani (Columbia University/New York Genome Center, United States)
4:30 pm - 6:00 pm
Poster Session
6:00 pm - 8:00 pm
Contribution of Aging to Neurodegenerative Disease
Discussion Leader: Hemali Phatnani (Columbia University/New York Genome Center, United States)
6:00 pm - 6:20 pm
Giovanna Mallucci (Altos Labs, United Kingdom)
"Boosting Resilience to Prevent Neurodegeneration"
6:20 pm - 6:30 pm
Discussion
6:30 pm - 6:50 pm
Catherine Kaczorowski (University of Michigan, United States)
"Using Complex Genetics in Mice to Unlock the Secrets of Cognitive Resilience"
6:50 pm - 7:00 pm
Discussion
7:00 pm - 7:20 pm
Ian Guldner (Stanford University, United States)
"Age-Related Protein Turnover Aberrations Revealed by New Protein Tagging Models"
7:20 pm - 7:30 pm
Discussion
7:30 pm - 7:40 pm
Gavin Wang (Medical University of South Carolina, United States)
"Role of Cellular Senescence in Brain Aging and Alzheimer’s Disease"
7:40 pm - 7:45 pm
Discussion
7:45 pm - 7:55 pm
Mathieu Bourdenx (University College London, United Kingdom)
"Contribution of Aging to Neurodegenerative Disease"
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
Frontotemporal Dementia and ALS Mechanistic Updates
Discussion Leader: Christian Haass (Ludwig Maximilians University of Munich, Germany)
9:00 am - 9:05 am
Introduction by Discussion Leader
9:05 am - 9:25 am
Michael Ward (National Institutes of Health, United States)
"Role for ANXA11in the Regulation of Lysosome-Associated Local Translation in Neurons"
9:25 am - 9:35 am
Discussion
9:35 am - 9:55 am
Philip Wong (Johns Hopkins Medicine, United States)
"TDP-43: Pathogenic Mechanism, Presymptomatic Fluid Biomarker and Therapeutic Strategy"
9:55 am - 10:05 am
Discussion
10:05 am - 10:15 am
Akshay Bhinge (Living Systems Institute, University of Exeter, United Kingdom)
"Elucidating Key Drivers of RNA Dysregulation in ALS Using a Novel Model of TDP43 Proteinopathy and ePRINT Technology"
10:15 am - 10:20 am
Discussion
10:20 am - 10:30 am
Gwyneth Welch (Dewpoint Therapeutics, United States)
"TDP-43 Condensate Modulation Rescues TDP-43 Loss of Function in ALS Patient-Derived Motor Neurons and Mouse Models of TDP-43 Proteinopathy"
10:30 am - 10:35 am
Discussion
10:35 am - 11:00 am
Coffee Break
11:00 am - 11:20 am
Jan Veldink (Department of Neurology, University Medical Center Utrecht, The Netherlands, The Netherlands)
"How DNA Research in All ALS Patients Will Change the Course of the Disease"
11:20 am - 11:30 am
Discussion
11:30 am - 11:40 am
Junjie Guo (Yale University, United States)
"Aberrant Splicing Exonizes C9ORF72 Repeat Expansion in ALS/FTD"
11:40 am - 11:45 am
Discussion
11:45 am - 11:55 am
Pete Harley (University College London, United Kingdom)
"The ALS-FTD Cryptic Exon in UNC13A Mediates Synaptic Dysfunction Following Loss of TDP-43"
11:55 am - 12:00 pm
Discussion
12:00 pm - 12:10 pm
Stephan Tetter (MRC Laboratory of Molecular Biology, United Kingdom)
"What’s the FUS About?"
12:10 pm - 12:15 pm
Discussion
12:15 pm - 12:25 pm
Diana Arseni (MRC Laboratory of Molecular Biology, United Kingdom)
"ABC of TDP-43"
12:25 pm - 12:30 pm
Discussion
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
Parkinson’s Disease: Mechanistic Updates
Discussion Leader: Mark Cookson (National Institute on Aging, NIH, United States)
6:00 pm - 6:20 pm
Sonia Gandhi (UCL & The Francis Crick Institute, United Kingdom)
"Single Cell and Single Molecule Approaches to Map Early Mechanisms in Parkinson's Brain and Cell Models"
6:20 pm - 6:30 pm
Discussion
6:30 pm - 6:50 pm
Tim Bartels (University College London / Dementia Research Institute, United Kingdom)
"Peripheral Immune Modulators in Body-First Parkinson’s Disease"
6:50 pm - 7:00 pm
Discussion
7:00 pm - 7:10 pm
Angelika Harbauer (MPI for Biological Intelligence, Germany)
"PINK1 Protein Biogenesis in Neurons Requires Integration of Signals from the ER and Endolysosomes to Enable Mitophagy"
7:10 pm - 7:15 pm
Discussion
7:15 pm - 7:35 pm
Emil Gustavsson (University College London, United Kingdom)
"Utilizing Long-Read RNA Sequencing to Enhance Insight and Improve Therapeutic Targets for Neurodegenerative Disease-Linked Loci"
7:35 pm - 7:45 pm
Discussion
7:45 pm - 7:55 pm
Alexei Pushechnikov (Expert Systems Inc., United States)
"Discovery of a Highly Selective, Orally Bioavailable, Brain-Penetrant LRRK2 Inhibitor"
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
Alzheimer’s Disease: Mechanistic Updates
Discussion Leader: Bart De Strooper (VIB and KU Leuven, Belgium)
9:00 am - 9:05 am
Introduction by Discussion Leader
9:05 am - 9:25 am
Rene Frank (University of Leeds, United Kingdom)
"In-Tissue Molecular Architecture in Alzheimer's Disease Post-mortem Brain"
9:25 am - 9:35 am
Discussion
9:35 am - 9:55 am
Christian Haass (Ludwig Maximilians University of Munich, Germany)
"Cellular Mechanisms of Late-Onset Alzheimer’s Disease Risk Variants"
9:55 am - 10:05 am
Discussion
10:05 am - 10:15 am
Kate Onuska (Western University, Canada)
"Molecular Cholinergic Deficits Drive Cortical Pathology in Presymptomatic Alzheimer’s Disease (PET Imaging)"
10:15 am - 10:20 am
Discussion
10:20 am - 10:30 am
Kai-Wen He (IRCBC, Shanghai Institute of Organic Chemistry ,Chinese Academy of Science, China)
"Brain Region-Specific Tau Hyperphosphorylation Mediates Alzheimer’s Disease-Associated Early-Onset Sleep Disruption"
10:30 am - 10:35 am
Discussion
10:35 am - 11:00 am
Coffee Break
11:00 am - 11:20 am
David Holtzman (Washington University in St. Louis, United States)
"Role of APOE and the Innate and Adaptive Immune Response in the Setting of Amyloid and Tau Pathology"
11:20 am - 11:30 am
Discussion
11:30 am - 11:50 am
Soyon Hong (University College London, United Kingdom)
"Understanding Neuro-Glia-Immune Crosstalk at the Vulnerable Synapse"
11:50 am - 12:00 pm
Discussion
12:00 pm - 12:20 pm
Michael Heneka (Université du Luxembourg, Luxembourg)
"Functional and Structural Impact of NLRP3 and NLRP1 Inflammasome Activation on Alzheimer Disease Pathogenesis"
12:20 pm - 12:30 pm
Discussion
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
Tauopathies: Mechanistic Updates
Discussion Leader: David Holtzman (Washington University in St. Louis, United States)
6:00 pm - 6:20 pm
Li Gan (Weill Cornell Medicine, United States)
"Uncovering Mechanisms of Tauopathy in Human iPSC Model"
6:20 pm - 6:30 pm
Discussion
6:30 pm - 6:50 pm
Martin Kampmann (University of California, San Francisco, United States)
"Mechanisms Controlling Cell Type-Selective Vulnerability to Tau Aggregation and Toxicity"
6:50 pm - 7:00 pm
Discussion
7:00 pm - 7:10 pm
Courtney Lane-Donovan (UCSF, United States)
"Tau Phosphorylation at Alzheimer’s Disease Biomarker Sites Impairs Tau Cleavage by Lysosomal Proteases"
7:10 pm - 7:15 pm
Discussion
7:15 pm - 7:25 pm
Jonathan Farley (Alnylam Pharmaceuticals, United States)
"RNAi Knockdown of Microtubule-Associated Protein Tau as a Therapeutic Strategy for Tauopathies"
7:25 pm - 7:30 pm
Discussion
7:30 pm - 7:40 pm
Miwei Hu (Washington University in St. Louis, United States)
"Antisense Oligonucleotide Development for 4R Tau Mutations: Evaluating Phenotypic and Sleep Changes in Tauopathy Models"
7:40 pm - 7:45 pm
Discussion
7:45 pm - 7:55 pm
Sumi Bez (UK Dementia Research Institute at UCL, United Kingdom)
"TRIM11 is a Novel E3 Ligase for Tau"
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; Election of the Next Vice Chair(s)
9:00 am - 12:30 pm
Biomarkers, Theranostics and Models
Discussion Leader: Stephanie Fradette (Biogen, United States)
9:00 am - 9:05 am
Introduction by Discussion Leader
9:05 am - 9:35 am
Henrik Zetterberg (UCL, Sweden)
"Biomarkers in Neurodegenerative Diseases"
9:35 am - 9:50 am
Discussion
9:50 am - 10:10 am
Elisa Konofagou (Columbia University, United States)
"Ultrasound-Mediated Immunomodulation and Drug Delivery in Neurodegerative Disease"
10:10 am - 10:20 am
Discussion
10:20 am - 10:30 am
Chia-Yi Kuan (University of Virginia School of Medicine, United States)
"[18F]Fluoroedaravone: a Positron Emission Tomography Probe for Imaging Oxidative Stress in the Central Nervous System"
10:30 am - 10:35 am
Discussion
10:35 am - 11:00 am
Coffee Break
11:00 am - 11:20 am
Andrew Yoo (Washington University School of Medicine, United States)
"Modeling Age-Associated Neurodegeneration of Late-Onset Disorders via Direct Neuronal Reprogramming"
11:20 am - 11:30 am
Discussion
11:30 am - 11:40 am
Martha Foiani (UK Dementia Research Institute at University College London, United Kingdom)
"Mutation-Dependent Phenotype in Novel Human Tau KI Mouse Models"
11:40 am - 11:45 am
Discussion
11:45 am - 11:55 am
Hubert Aviolat (AbbVie, Germany)
"Highly Sensitive Ligand-Binding Assay Confirms DOPA Decarboxylase in Plasma and Cerebrospinal Fluid as a Promising Biomarker Candidate for Parkinson's Disease"
11:55 am - 12:00 pm
Discussion
12:00 pm - 12:10 pm
Hayk Davtyan (University of California, Irvine, United States)
"Brain-Wide Engraftment of Human Monocytes, but Not Microglia, Induces a Chronic Proinflammatory State, Promoting Synaptic and Behavioral Dysfunction"
12:10 pm - 12:15 pm
Discussion
12:15 pm - 12:25 pm
Benjamin Tuck (Astex Therapeutics, United Kingdom)
"Development of a Comprehensive Cellular Platform to Study Lysosomal Dynamics for Neurodegenerative Disease Research"
12:25 pm - 12:30 pm
Discussion
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
Novel Therapeutic Approaches
Discussion Leader: Eric Karran (AbbVie Inc, United States)
6:00 pm - 6:20 pm
Sheena Chew (Biogen, United States)
"The ALSpire Study: A Clinical Trial of an Antisense Oligonucleotide Targeting ATXN2 in Adults with ALS"
6:20 pm - 6:30 pm
Discussion
6:30 pm - 6:50 pm
Stefan Pulst (University of Utah, United States)
"Targeting the ATXN2/STAU1 Complex in Cerebellar and Motor Neuron Degeneration"
6:50 pm - 7:00 pm
Discussion
7:00 pm - 7:20 pm
Adrian Krainer (Cold Spring Harbor Laboratory, United States)
"Advances in Splice-Correction Therapies for Spinal Muscular Atrophy"
7:20 pm - 7:30 pm
Discussion
7:30 pm - 7:50 pm
Miranda Orr (Wake Forest University School of Medicine, United States)
"Senescent Cells as a Therapeutic Target for Neurodegenerative Diseases"
7:50 pm - 8:00 pm
Discussion
8:00 pm - 9:00 pm
Dinner
Friday
7:30 am - 8:30 am
Breakfast
9:00 am
Departure