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
Motor Neuron Gene Discovery
Discussion Leader: Bryan Traynor (National Institutes of Health, USA)
7:40 pm - 8:20 pm
David Goldstein (Columbia University Medical Center , USA)
"Genomics and Precision Medicine in ALS"
8:20 pm - 8:35 pm
Discussion
8:35 pm - 9:15 pm
John Landers (University of Massachusetts Medical School, USA)
"ALS Genetics: Where Are We Now and Where Are We Going?"
9:15 pm - 9:30 pm
Discussion
Monday
7:30 am - 8:30 am
Breakfast
8:30 am - 9:00 am
Group Photo
9:00 am - 12:30 pm
In Vivo Models of Motor Neuron Diseases
Discussion Leader: Don Cleveland (Ludwig Institute for Cancer Research, USA)
9:00 am - 9:35 am
Cat Lutz (The Jackson Laboratory, USA)
"Mouse Models for ALS, New and Old"
9:35 am - 9:45 am
Discussion
9:45 am - 10:20 am
Laura Ranum (University of Florida College of Medicine, USA)
"C9ORF72 ALS/FTD: Molecular Insights and Therapeutic Efforts to Block Paralysis and Neurodegeneration in C9-BAC Transgenic Mice"
10:20 am - 10:30 am
Discussion
10:30 am - 11:00 am
Coffee Break
11:00 am - 11:35 am
Robert Baloh (Cedars-Sinai Medical Center, USA)
"Role of C9orf72 in Neurodegeneration and Neuroinflammation"
11:35 am - 11:45 am
Discussion
11:45 am - 11:55 am
Selected from Poster Abstracts: Aaron Burberry (Harvard University, USA)
"A New Mouse Model of C9ORF72-ALS Containing both Gain and Loss of Function Mutations"
11:55 am - 12:00 pm
Discussion
12:00 pm - 12:10 pm
Selected from Poster Abstracts: Valeria Gerbino (Columbia University, USA)
"Loss of TBK1 Affects Motor Neuron Autophagy and Disease Progression in a Mouse Model of ALS"
12:10 pm - 12:15 pm
Discussion
12:15 pm - 12:25 pm
Selected from Poster Abstracts: Daniela Zarnescu (University of Arizona, USA)
"Deciphering Translation Dysregulation Mechanisms Across Multiple Models of ALS"
12:25 pm - 12:30 pm
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
RNA Granules and Proteinopathies in ALS
Discussion Leader: Christopher Donnelly (University of Pittsburgh, USA)
7:30 pm - 8:00 pm
Peter St George-Hyslop (University of Toronto, Canada)
"Modulation of Phase Transition of RNA Binding Proteins in Regulating Protein Expression in Neuronal Termini"
8:00 pm - 8:10 pm
Discussion
8:10 pm - 8:40 pm
Simon Alberti (Max Planck Institute of Molecular Cell Biology and Genetics, Germany)
"RNP Granules: How They Form, Age and Cause Disease"
8:40 pm - 8:50 pm
Discussion
8:50 pm - 9:20 pm
Davide Trotti (Thomas Jefferson University, USA)
"Propagation of Disease Linked Poly-Dipeptides in C9orf72-FTD/ALS"
9:20 pm - 9:30 pm
Discussion
Tuesday
7:30 am - 8:30 am
Breakfast
9:00 am - 12:30 pm
Dipeptide and RNA-Mediated Toxicity to Motor Neurons
Discussion Leader: Dieter Edbauer (German Center for Neurodegenerative Diseases (DZNE), Germany)
9:00 am - 9:35 am
Andre Hoelz (California Institute of Technology, USA)
"Structure of the Nuclear Pore Complex"
9:35 am - 9:45 am
Discussion
9:45 am - 10:20 am
James Manley (Columbia University, USA)
"Mechanisms of Gene Expression Dysregulation in ALS"
10:20 am - 10:30 am
Discussion
10:30 am - 11:00 am
Coffee Break
11:00 am - 11:35 am
Justin Ichida (University of Southern California, USA)
"Identification of Therapeutic Targets for ALS Using Phenotypic Chemical and Genetic Screens on Patient-Derived Neurons"
11:35 am - 11:45 am
Discussion
11:45 am - 11:55 am
Joshua Wheeler (University of Colorado, Boulder, USA)
"Amyloids in Skeletal Muscle"
11:55 am - 12:00 pm
Discussion
12:00 pm - 12:10 pm
Selected from Poster Abstracts: Anna Corrionero (Massachusetts Institute of Technology / Howard Hughes Medical Institute, USA)
"A C9orf72 ALS/FTD Ortholog Acts in Endolysosomal Degradation and Lysosomal Homeostasis"
12:10 pm - 12:15 pm
Discussion
12:15 pm - 12:25 pm
Aaron Haeusler (Thomas Jefferson University, USA)
"Modeling Therapeutic Opportunities for Attenuating C9orf72 NRE-Linked Disease"
12:25 pm - 12:30 pm
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
Emerging Biomarkers of Motor Neuron Disease
Discussion Leader: Nicholas Maragakis (Johns Hopkins University, USA)
7:30 pm - 8:05 pm
Frank Bennett (Ionis Pharmaceuticals, USA)
"Antisense Oligonucleotide Therapeutics for Neuromuscular Diseases"
8:05 pm - 8:15 pm
Discussion
8:15 pm - 8:50 pm
Martin Turner (University of Oxford, United Kingdom)
"System Versus Cell: Pathophysiological Insights from Brain Neuroimaging"
8:50 pm - 9:00 pm
Discussion
9:00 pm - 9:10 pm
Selected from Poster Abstracts: Eran Hornstein (Weizmann Institute of Science, Israel)
"miRNA Failure in ALS"
9:10 pm - 9:15 pm
Discussion
9:15 pm - 9:25 pm
Selected from Poster Abstracts: Kaitlin Weskamp (University of Michigan, USA)
"Activity-Dependent Changes in TDP43 Autoregulation and Neuronal Survival in ALS Models"
9:25 pm - 9:30 pm
Discussion
Wednesday
7:30 am - 8:30 am
Breakfast
9:00 am - 12:30 pm
Decoding the Role of TDP-43 and FUS in Neurodegeneration
Discussion Leader: Philip Wong (Johns Hopkins University, USA)
9:00 am - 9:35 am
Sami Barmada (University of Michigan, USA)
"Single-Cell TDP43 Homeostasis in Models of Amyotrophic Lateral Sclerosis"
9:35 am - 9:45 am
Discussion
9:45 am - 10:20 am
James Shorter (University of Pennsylvania, USA)
"Reversing Aberrant Phase Transitions Connected to ALS"
10:20 am - 10:30 am
Discussion
10:30 am - 11:00 am
Coffee Break
11:00 am - 11:35 am
Clotilde Lagier-Tourenne (Harvard Medical School / Massachusetts General Hospital, USA)
"Modeling C9ORF72 Disease: A Crucial Step for Therapeutic Development in ALS/FTD"
11:35 am - 11:45 am
Discussion
11:45 am - 11:55 am
Selected from Poster Abstracts: John Carson (University of Connecticut Health Center, USA)
"G4C2 Repeat RNA Activates PKR and Inhibits Translation in A2 RNA Granules"
11:55 am - 12:00 pm
Discussion
12:00 pm - 12:10 pm
Selected from Poster Abstracts: Udai Pandey (University of Pittsburgh Medical Center, USA)
"Mucleblind Protects Against Neurodegeneration Caused by Pathogenic Mutations in FUS"
12:10 pm - 12:15 pm
Discussion
12:15 pm - 12:25 pm
Selected from Poster Abstracts: Carlos Castaneda (Syracuse University, USA)
"Liquid-Liquid Phase Separation of ALS-Linked UBQLN2 Is Modulated by Oligomerization and Ubiquitin Binding"
12:25 pm - 12:30 pm
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
Motor Circuits and Cell Based Systems
Discussion Leader: Fen-Biao Gao (University of Massachusetts Medical School, USA)
7:30 pm - 8:05 pm
Siddharthan Chandran (University of Edinburgh, United Kingdom)
"Neuron-Glial Models Systems for Neurodegeneration"
8:05 pm - 8:15 pm
Discussion
8:15 pm - 8:50 pm
George Mentis (Columbia University, USA)
"Imbalance Between Excitatory and Inhibitory Neurotransmission Causes Motor Neuron Dysfunction in Spinal Muscular Atrophy"
8:50 pm - 9:00 pm
Discussion
9:00 pm - 9:10 pm
Selected from Poster Abstracts: Ashley Boehringer (Barrow Neurological Institute, USA)
"ALS Associated Mutations in Matrin 3 Alter Protein-Protein Interactions and Impede mRNA Nuclear Export"
9:10 pm - 9:15 pm
Discussion
9:15 pm - 9:25 pm
Selected from Poster Abstracts: Ke Zhang (Johns Hopkins University, USA)
"Ataxin-2 in Stress Granules Regulates Nucleocytoplasmic Transport in ALS"
9:25 pm - 9:30 pm
Discussion
Thursday
7:30 am - 8:30 am
Breakfast
9:00 am - 12:30 pm
Axons and Glial Cells
Discussion Leader: Richard Ransohoff (Biogen, USA)
9:00 am - 9:35 am
Beth Stevens (Boston Children's Hospital, USA)
"How Microglia Prune Synapses in Health and Disease"
9:35 am - 9:45 am
Discussion
9:45 am - 10:20 am
Erika Holzbaur (University of Pennsylvania, USA)
"Autophagy Dynamics in Neuronal Homeostasis and Neurodegeneration"
10:20 am - 10:30 am
Discussion
10:30 am - 11:00 am
Coffee Break
11:00 am - 11:35 am
Richard Robitaille (University of Montreal, Canada)
"Glial Mismanagement of Neuromuscular Junction Structure and Function in ALS"
11:35 am - 11:45 am
Discussion
11:45 am - 11:55 am
Selected from Poster Abstracts: Karen Ling (Ionis Pharmaceuticals, Inc, USA)
"Evaluating Epha4 as a Therapeutic Target for Amyotrophic Lateral Sclerosis by Antisense-Mediated Inhibition in the Adult Mouse CNS"
11:55 am - 12:00 pm
Discussion
12:00 pm - 12:10 pm
Selected from Poster Abstracts: Krista Spiller (Center for Neurodegenerative Disease Research, University of Pennsylvania, USA)
"Microglia-Mediated Recovery from Motor Neuron Degeneration"
12:10 pm - 12:15 pm
Discussion
12:15 pm - 12:25 pm
Selected from Poster Abstracts: Jonathan Grima (Johns Hopkins University, USA)
"The Nuclear Pore Complex Is Compromised in ALS"
12:25 pm - 12:30 pm
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
From Big Data Analysis to Therapeutics
Discussion Leader: Kenneth Fischbeck (National Institute of Neurological Disorders and Stroke, NIH, USA)
7:30 pm - 8:00 pm
Ernest Fraenkel (Massachusetts Institute of Technology, USA)
"Integrating Transcriptional, Epigenomic and Proteomic Data to Reveal Pathways Altered by Mutated C9ORF72 in iPSC-Derived Motor Neurons"
8:00 pm - 8:10 pm
Discussion
8:10 pm - 8:40 pm
Leonard Petrucelli (Mayo Clinic College of Medicine and Science, USA)
"Mechanistic and Therapeutic Insights into C9ALS"
8:40 pm - 8:50 pm
Discussion
8:50 pm - 9:20 pm
Matthew Disney (The Scripps Research Institute, USA)
"Design of Small Molecules Targeting Repeating Transcripts"
9:20 pm - 9:30 pm
Discussion
Friday
7:30 am - 8:30 am
Breakfast
9:00 am
Departure