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 Site Staff / Welcome from the GRC Chair
7:40 pm - 9:30 pm
Clinical Phenotypes in ALS and Related Motor Neuron Diseases
Discussion Leader: Jeffrey Rothstein (Johns Hopkins University, USA)
7:40 pm - 7:50 pm
Opening Remarks
7:50 pm - 8:15 pm
John Ravits (University of California, San Diego, USA)
"Neuroanatomic Neuropathologic Correlations with ALS Phenotypes"
8:15 pm - 8:20 pm
Discussion
8:20 pm - 8:45 pm
Thomas Crawford (Johns Hopkins University, USA)
"Current and Future Therapeutic Interventions in SMA"
8:45 pm - 8:50 pm
Discussion
8:50 pm - 9:15 pm
Merit Cudkowicz (Massachusetts General Hospital, USA)
"Accelerating Therapy Development: Master Platform Trial Initiatives and Subgroups"
9:15 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
Axonal Biology and Motor Neuron Loss
Discussion Leader: Giampietro Schiavo (University College London, United Kingdom)
9:00 am - 9:25 am
Wilfried Rossoll (Mayo Clinic, USA)
"Nuclear Transport Receptors as Regulators of Protein Aggregation and Toxicity in ALS"
9:25 am - 9:30 am
Discussion
9:30 am - 9:55 am
Jeffery Twiss (University of South Carolina, USA)
"Regulation of Axonal Protein Synthesis by G3BP1 Aggregation"
9:55 am - 10:00 am
Discussion
10:00 am - 10:25 am
Ludo Van Den Bosch (KU Leuven / VIB, Belgium)
"Axonal Transport Defects in iPSC-Derived Motor Neurons"
10:25 am - 10:30 am
Discussion
10:30 am - 11:00 am
Coffee Break
11:00 am - 11:25 am
Michael Ward (National Institutes of Health, USA)
"A Surprising Link Between Lysosomal and RNA Biology in ALS Pathogenesis"
11:25 am - 11:30 am
Discussion
11:30 am - 11:55 am
Selected from Poster Abstracts: Pietro Fratta (University College London, United Kingdom)
"ALS FUS Mutations Alter Splicing and Translation Through Loss and Gain of Function"
11:55 am - 12:00 pm
Discussion
12:00 pm - 12:10 pm
Selected from Poster Abstracts: Jik Nijssen (Karolinska Institutet, Sweden)
"Decoding Motor Axon Biology in Health and ALS Using Axon-Seq"
12:10 pm - 12:15 pm
Discussion
12:15 pm - 12:30 pm
General Discussion
12:30 pm - 1:30 pm
Lunch
1:30 pm - 4:00 pm
Free Time
3:00 pm - 4:00 pm
The GRC Power Hourâ„¢
The GRC Power Hourâ„¢ is designed to address challenges women face in science and issues of diversity and inclusion. The program supports the professional growth of all members of our communities by providing an open forum for discussion and mentoring.
Organizer: Daniela Zarnescu (University of Arizona, USA)
4:00 pm - 6:00 pm
Poster Session
6:00 pm - 7:00 pm
Dinner
7:30 pm - 9:30 pm
Role of RNA Binding Proteins in Motor Neuron Degeneration
Discussion Leader: Laura Ranum (University of Florida College of Medicine, USA)
7:30 pm - 7:55 pm
Don Cleveland (Ludwig Institute for Cancer Research, USA)
"Therapy Development for Disease from TDP-43 Loss and/or Gain-of-Function"
7:55 pm - 8:00 pm
Discussion
8:00 pm - 8:25 pm
Magdalini Polymenidou (University of Zurich, Switzerland)
"Pathological TDP-43 Assemblies and Their Link to Disease Heterogeneity"
8:25 pm - 8:30 pm
Discussion
8:30 pm - 8:55 pm
Chris Shaw (King's College London, United Kingdom)
"ALS and RNA: Thinking Outside the Nuclear Box"
8:55 pm - 9:00 pm
Discussion
9:00 pm - 9:10 pm
Selected from Poster Abstracts: Kazuhide Asakawa (Tokyo Medical University / National Institute of Genetics, Japan)
"Pathological Alterations of the Spinal Motor Neurons Induced by Optogenetic TDP-43 Oligomerization"
9:10 pm - 9:15 pm
Discussion
9:15 pm - 9:25 pm
Selected from Poster Abstracts: Veronica Ryan (Brown University, USA)
"Phosphorylation Disrupts hnRNPA2 Aggregation and Alters Interactions with mRNA Transport Granule Components"
9:25 pm - 9:30 pm
Discussion
Tuesday
7:30 am - 8:30 am
Breakfast
8:30 am - 9:00 am
Group Photo
9:00 am - 12:30 pm
Mechanisms of Aberrant RNA Metabolism
Discussion Leader: Sami Barmada (University of Michigan, USA)
9:00 am - 9:25 am
Fen-Biao Gao (University of Massachusetts Medical School, USA)
"Production and Toxicity of Poly(GR) in C9ORF72-ALS/FTD"
9:25 am - 9:30 am
Discussion
9:30 am - 9:55 am
Jeffrey Rothstein (Johns Hopkins University, USA)
"Disassembly and Reassembly of the Nuclear Pore Complex in ALS/FTD"
9:55 am - 10:00 am
Discussion
10:00 am - 10:25 am
Charlotte Sumner (Johns Hopkins University School of Medicine, USA)
"Therapeutic Targeting of RNA in Spinal Muscular Atrophy"
10:25 am - 10:30 am
Discussion
10:30 am - 11:00 am
Coffee Break
11:00 am - 11:25 am
Dorothee Dormann (Ludwig Maximilian University of Munich, Germany)
"Molecular Mechanisms Controlling Localization and Phase Separation of ALS-Linked RNA-Binding Proteins"
11:25 am - 11:30 am
Discussion
11:30 am - 11:55 am
Selected from Poster Abstracts: Udai Pandey (University of Pittsburgh Medical Center, USA)
"Pathogenic Mutations in Gemin5 Cause Early-Onset Motor Dysfunction and Cerebellar Atrophy and Disrupt snRNP Complex"
11:55 am - 12:00 pm
Discussion
12:00 pm - 12:10 pm
Selected from Poster Abstracts: Stephen Moore (Barrow Neurological Institute, USA)
"ADAR2 Mislocalization and Widespread RNA Editing Aberrations in C9orf72-Mediated ALS/FTD"
12:10 pm - 12:15 pm
Discussion
12:15 pm - 12:30 pm
General Discussion
12:30 pm - 1:30 pm
Lunch
1:30 pm - 4:00 pm
Free Time
4:00 pm - 6:00 pm
Poster Session
6:00 pm - 7:00 pm
Dinner
7:30 pm - 9:30 pm
Role of Proteinopathies in Motor Neuron Disease
Discussion Leader: David Borchelt (University of Florida, USA)
7:30 pm - 7:55 pm
Albert La Spada (Duke University, USA)
"Cellular and Molecular Basis of Motor Neuron Demise: From SBMA to ALS"
7:55 pm - 8:00 pm
Discussion
8:00 pm - 8:25 pm
Diane Merry (Thomas Jefferson University, USA)
"Exploiting the Androgen Receptor Interactome to Identify Pathogenic Mechanisms in SBMA"
8:25 pm - 8:30 pm
Discussion
8:30 pm - 8:55 pm
Serena Carra (University of Modena and Reggio Emilia, Italy)
"Protein Quality Control of Membraneless Organelles: Implications in ALS and Neurodegenerative Diseases"
8:55 pm - 9:00 pm
Discussion
9:00 pm - 9:10 pm
Selected from Poster Abstracts: Nicolas Gomez (University of Michigan, USA)
"Investigating the Mechanisms of UPF1-Mediated Rescue of TDP-43 Toxicity"
9:10 pm - 9:15 pm
Discussion
9:15 pm - 9:25 pm
Selected from Poster Abstracts: Joseph Klim (Harvard University, USA)
"ALS-Implicated Protein TDP-43 Sustains Levels of STMN2, a Mediator of Motor Neuron Growth and Regeneration"
9:25 pm - 9:30 pm
Discussion
Wednesday
7:30 am - 8:30 am
Breakfast
9:00 am - 12:30 pm
Aberrant Protein Control and Related Mechanisms
Discussion Leader: Heather Durham (McGill University, Canada)
9:00 am - 9:25 am
F. Ulrich Hartl (Max Planck Institute of Biochemistry, Germany)
"The Nucleolus as a Protein Quality Control Compartment"
9:25 am - 9:30 am
Discussion
9:30 am - 9:55 am
Mondira Kundu (St. Jude Children's Research Hospital, USA)
"Role of the Autophagy Inducing Kinases, ULK1 and ULK2, in Degenerative Disorders"
9:55 am - 10:00 am
Discussion
10:00 am - 10:25 am
Christine Vande Velde (Centre de Recherche du CHUM, Universite de Montreal, Canada)
"Loss of Nuclear TDP-43 Function as a Mechanism of ALS Pathogenesis"
10:25 am - 10:30 am
Discussion
10:30 am - 11:00 am
Coffee Break
11:00 am - 11:25 am
Wade Harper (Harvard Medical School, USA)
"Linking Selective Autophagy to Neurodegenerative Disease"
11:25 am - 11:30 am
Discussion
11:30 am - 11:55 am
Selected from Poster Abstracts: Michael Hughes (University of California, Los Angeles, USA)
"Latest Insights from Amyloid Structures: Steric Zippers and LARKS in Fibrils"
11:55 am - 12:00 pm
Discussion
12:00 pm - 12:10 pm
Selected from Poster Abstracts: Evan Lester (University of Colorado Boulder, USA)
"The RNA Composition of Tau Aggregates in a Cellular Model of FTD"
12:10 pm - 12:15 pm
Discussion
12:15 pm - 12:30 pm
General Discussion
12:30 pm - 1:30 pm
Lunch
1:30 pm - 4:00 pm
Free Time
4:00 pm - 6:00 pm
Poster Session
6:00 pm - 7:00 pm
Dinner
7:00 pm - 7:30 pm
Business Meeting
Nominations for the Next Vice Chair; Fill in Conference Evaluation Forms; Discuss Future Site and Scheduling Preferences; Election of the Next Vice Chair
7:30 pm - 9:30 pm
Role of Neuroinflammation in Neurodegeneration
Discussion Leader: Robert Baloh (Cedars-Sinai Medical Center, USA)
7:30 pm - 7:55 pm
Molly Hammell (Cold Spring Harbor Laboratory, USA)
"Three Subtypes of ALS: Retrotransposons, Stress and Inflammation"
7:55 pm - 8:00 pm
Discussion
8:00 pm - 8:25 pm
Eric Huang (University of California, San Francisco, USA)
"Microglial Toxicity and TDP-43 Proteinopathy in Neurodegeneration"
8:25 pm - 8:30 pm
Discussion
8:30 pm - 8:55 pm
Junying Yuan (Harvard Medical School, USA)
"The Role and Mechanism of RIPK1 in Mediating Neuroinflammation and Cell Death in ALS"
8:55 pm - 9:00 pm
Discussion
9:00 pm - 9:10 pm
Selected from Poster Abstracts: Aaron Burberry (Harvard University, USA)
"C9orf72 Functions to Suppress Systemic and Neural Inflammation Induced by Gut Microbiota"
9:10 pm - 9:15 pm
Discussion
9:15 pm - 9:25 pm
Selected from Poster Abstracts: Jochen Weishaupt (Ulm University, Germany)
"Effects of Heterozygous Tbk1 Deletion in Old or Mutant SOD1 Transgenic Mice"
9:25 pm - 9:30 pm
Discussion
Thursday
7:30 am - 8:30 am
Breakfast
9:00 am - 12:30 pm
Glial Biology of Motor Neuron Diseases
Discussion Leader: Davide Trotti (Thomas Jefferson University, USA)
9:00 am - 9:25 am
Shane Liddelow (NYU Langone Medical Center, USA)
"What Do Reactive Astrocytes (Actually) Do?"
9:25 am - 9:30 am
Discussion
9:30 am - 9:55 am
Nicholas Maragakis (Johns Hopkins University, USA)
"Astrocyte Hemichannels as Conduits for Motor Neuron Death in ALS"
9:55 am - 10:00 am
Discussion
10:00 am - 10:25 am
Dwight Bergles (Johns Hopkins University School of Medicine, USA)
"Stage Specific Control of Oligodendrocytes by TDP-43"
10:25 am - 10:30 am
Discussion
10:30 am - 11:00 am
Coffee Break
11:00 am - 11:25 am
Tara Spires-Jones (The University of Edinburgh, United Kingdom)
"Glial Involvement in Synapse Loss in Alzheimer's Disease and ALS: Evidence from Human Post-Mortem Brain"
11:25 am - 11:30 am
Discussion
11:30 am - 11:40 am
Selected from Poster Abstracts: Deepti Lall (Cedars-Sinai Medical Center, USA)
"C9orf72 Deficiency Promotes Microglial Mediated Synaptic Loss in Aging or Amyloid Accumulation"
11:40 am - 11:45 am
Discussion
11:45 am - 11:55 am
Selected from Poster Abstracts: Laura Ferraiuolo (Sheffield Institute for Translational Neuroscience, University of Sheffield, United Kingdom)
"Using Patient-Derived Astrocytes to Inform Precision Medicine Approaches"
11:55 am - 12:00 pm
Discussion
12:00 pm - 12:10 pm
Selected from Poster Abstracts: Marta Olah (Columbia University Medical Center, USA)
"Single Cell RNA Sequencing Uncovers Disease Associated Changes in the Population Structure of Human Microglia Specific to Amyotrophic Lateral Sclerosis"
12:10 pm - 12:15 pm
Discussion
12:15 pm - 12:30 pm
General Discussion
12:30 pm - 1:30 pm
Lunch
1:30 pm - 4:00 pm
Free Time
4:00 pm - 6:00 pm
Poster Session
6:00 pm - 7:00 pm
Dinner
7:30 pm - 9:30 pm
Experimental Therapeutics and Biomarkers
Discussion Leader: Holly Kordasiewicz (Ionis Pharmaceuicals, USA)
7:30 pm - 7:55 pm
Richard Finkel (Nemours Children's Hospital, USA)
"The New Frontier of Spinal Muscular Atrophy: When, Where and How to Mitigate a Neurodegenerative Disease"
7:55 pm - 8:00 pm
Discussion
8:00 pm - 8:25 pm
Timothy Miller (Washington University in St. Louis, USA)
"RNA-Targeted Mechanisms and Therapeutics for ALS"
8:25 pm - 8:30 pm
Discussion
8:30 pm - 8:55 pm
Daniel Tardiff (Yumanity Therapeutics, USA)
"Transforming Target Identification for Neurodegenerative Diseases: Proof of Concept in Synucleinopathies and Progress in ALS"
8:55 pm - 9:00 pm
Discussion
9:00 pm - 9:15 pm
General Discussion
9:15 pm - 9:30 pm
Closing Remarks
Friday
7:30 am - 8:30 am
Breakfast
9:00 am
Departure