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Amyotrophic Lateral Sclerosis (ALS) and Related Motor Neuron Diseases
Gordon Research Conference

From Pathways of Motor Neuron Degeneration to Interventional Strategies

Dates

July 23-28, 2017

Location

Stoweflake Conference Center
Stowe, VT

Organizers

Chairs:
Jeffrey D. Rothstein & Piera Pasinelli

Vice Chairs:
Rita Sattler & J. Paul Taylor

Meeting Description

The goal of this meeting is to provide a dynamic forum for the active exchange of new discoveries and research tools in the advancing field of motor neuron diseases. Motor neuron diseases are a family of inherited and sporadic diseases affecting children and adults, typically are progressive and often fatal. The last 5+ years has seen tremendous growth in the understanding and development of relevant models for motor neuron diseases, such as amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA). This reflects the discovery of disease causing gene mutations, multiple transgenic animal, fish and fly models and disease relevant human patient derived iPS cell models of brain neurons and glia. The field has seen a convergence of multiple different threads of basic and clinical science disciplines- basic synaptic circuitry, RNA processing, nuclear trafficking, neuron-glial interactions, to whole genome, transcriptome, epigenome and proteomic approaches to diverse pathways, clinical subtypes and disease relevant drug targeting pathways. More recently, even wearable devices monitoring health- are intersecting these basic biological approaches. The conference will bring together experts in a wide range of basic, clinical and translational neuroscience to discuss advances in the progress of understanding common and rare motor neuron diseases. Exciting new areas of research in these disease include dysfunction in RNA metabolism and protein homeostasis, with specific defects in nucleocytoplasmic trafficking, induction of endoplasmic reticulum stress, and impaired dynamics of ribonucleoprotein bodies such as RNA granules. In addition, the use and validation of imaging modalities, such as PET, as well as large population multi-omics data generation, to identify and validate dysfunctional pathways in animal models and patients, as well as the development of new therapies and biomarkers will be discussed. The extraordinary recent progress in understanding the biology of ALS provides new grounds for optimism that meaningful therapies for ALS will be identified. Recent studies indicate that pathways and drugs that may be relevant to these diseases, have far wider applicability to other neurological injuries.

Contributors

Final Meeting Program

Sunday
2:00 pm - 9:00 pmArrival and Check-in
6:00 pmDinner
7:30 pm - 7:40 pmWelcome / Introductory Comments by GRC Site Staff
7:40 pm - 9:30 pmMotor Neuron Gene Discovery
Discussion Leader: Bryan Traynor (National Institutes of Health, USA)
7:40 pm - 8:20 pmDavid Goldstein (Columbia University Medical Center, USA)
"Genomics and Precision Medicine in ALS"
8:20 pm - 8:35 pmDiscussion
8:35 pm - 9:15 pmJohn Landers (University of Massachusetts Medical School, USA)
"ALS Genetics: Where Are We Now and Where Are We Going?"
9:15 pm - 9:30 pmDiscussion
Monday
7:30 am - 8:30 amBreakfast
8:30 amGroup Photo
9:00 am - 12:30 pmIn Vivo Models of Motor Neuron Diseases
Discussion Leader: Don Cleveland (Ludwig Institute for Cancer Research, USA)
9:00 am - 9:35 amCat Lutz (The Jackson Laboratory, USA)
"Mouse Models for ALS, New and Old"
9:35 am - 9:45 amDiscussion
9:45 am - 10:20 amLaura 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 amDiscussion
10:30 am - 11:00 amCoffee Break
11:00 am - 11:35 amRobert Baloh (Cedars-Sinai Medical Center, USA)
"Role of C9orf72 in Neurodegeneration and Neuroinflammation"
11:35 am - 11:45 amDiscussion
11:45 am - 11:55 amSelected 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 pmDiscussion
12:00 pm - 12:10 pmSelected 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 pmDiscussion
12:15 pm - 12:25 pmSelected from Poster Abstracts: Daniela Zarnescu (University of Arizona, USA)
"Deciphering Translation Dysregulation Mechanisms Across Multiple Models of ALS"
12:25 pm - 12:30 pmDiscussion
12:30 pmLunch
1:30 pm - 4:00 pmFree Time
4:00 pm - 6:00 pmPoster Session
6:00 pmDinner
7:30 pm - 9:30 pmRNA Granules and Proteinopathies in ALS
Discussion Leader: Christopher Donnelly (University of Pittsburgh, USA)
7:30 pm - 8:00 pmPeter 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 pmDiscussion
8:10 pm - 8:40 pmSimon 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 pmDiscussion
8:50 pm - 9:20 pmDavide Trotti (Thomas Jefferson University, USA)
"Propagation of Disease Linked Poly-Dipeptides in C9orf72-FTD/ALS"
9:20 pm - 9:30 pmDiscussion
Tuesday
7:30 am - 8:30 amBreakfast
9:00 am - 12:30 pmDipeptide and RNA-Mediated Toxicity to Motor Neurons
Discussion Leader: Dieter Edbauer (German Center for Neurodegenerative Diseases (DZNE), Germany)
9:00 am - 9:35 amAndre Hoelz (California Institute of Technology, USA)
"Structure of the Nuclear Pore Complex"
9:35 am - 9:45 amDiscussion
9:45 am - 10:20 amJames Manley (Columbia University, USA)
"Mechanisms of Gene Expression Dysregulation in ALS"
10:20 am - 10:30 amDiscussion
10:30 am - 11:00 amCoffee Break
11:00 am - 11:35 amJustin 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 amDiscussion
11:45 am - 11:55 amJoshua Wheeler (University of Colorado, Boulder, USA)
"Amyloids in Skeletal Muscle"
11:55 am - 12:00 pmDiscussion
12:00 pm - 12:10 pmSelected 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 pmDiscussion
12:15 pm - 12:25 pmAaron Haeusler (Thomas Jefferson University, USA)
"Modeling Therapeutic Opportunities for Attenuating C9orf72 NRE-Linked Disease"
12:25 pm - 12:30 pmDiscussion
12:30 pmLunch
1:30 pm - 4:00 pmFree Time
4:00 pm - 6:00 pmPoster Session
6:00 pmDinner
7:30 pm - 9:30 pmEmerging Biomarkers of Motor Neuron Disease
Discussion Leader: Nicholas Maragakis (Johns Hopkins University, USA)
7:30 pm - 8:05 pmFrank Bennett (Ionis Pharmaceuticals, USA)
"Antisense Oligonucleotide Therapeutics for Neuromuscular Diseases"
8:05 pm - 8:15 pmDiscussion
8:15 pm - 8:50 pmMartin Turner (University of Oxford, United Kingdom)
"System Versus Cell: Pathophysiological Insights from Brain Neuroimaging"
8:50 pm - 9:00 pmDiscussion
9:00 pm - 9:10 pmSelected from Poster Abstracts: Eran Hornstein (Weizmann Institute of Science, Israel)
"miRNA Failure in ALS"
9:10 pm - 9:15 pmDiscussion
9:15 pm - 9:25 pmSelected 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 pmDiscussion
Wednesday
7:30 am - 8:30 amBreakfast
9:00 am - 12:30 pmDecoding the Role of TDP-43 and FUS in Neurodegeneration
Discussion Leader: Philip Wong (Johns Hopkins University, USA)
9:00 am - 9:35 amSami Barmada (University of Michigan, USA)
"Single-Cell TDP43 Homeostasis in Models of Amyotrophic Lateral Sclerosis"
9:35 am - 9:45 amDiscussion
9:45 am - 10:20 amJames Shorter (University of Pennsylvania, USA)
"Reversing Aberrant Phase Transitions Connected to ALS"
10:20 am - 10:30 amDiscussion
10:30 am - 11:00 amCoffee Break
11:00 am - 11:35 amClotilde 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 amDiscussion
11:45 am - 11:55 amSelected 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 pmDiscussion
12:00 pm - 12:10 pmSelected 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 pmDiscussion
12:15 pm - 12:25 pmSelected 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 pmDiscussion
12:30 pmLunch
1:30 pm - 4:00 pmFree Time
4:00 pm - 6:00 pmPoster Session
6:00 pmDinner
7:00 pm - 7:30 pmBusiness 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 pmMotor Circuits and Cell Based Systems
Discussion Leader: Fen-Biao Gao (University of Massachusetts Medical School, USA)
7:30 pm - 8:05 pmSiddharthan Chandran (University of Edinburgh, United Kingdom)
"Neuron-Glial Models Systems for Neurodegeneration"
8:05 pm - 8:15 pmDiscussion
8:15 pm - 8:50 pmGeorge Mentis (Columbia University, USA)
"Imbalance Between Excitatory and Inhibitory Neurotransmission Causes Motor Neuron Dysfunction in Spinal Muscular Atrophy"
8:50 pm - 9:00 pmDiscussion
9:00 pm - 9:10 pmSelected 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 pmDiscussion
9:15 pm - 9:25 pmSelected from Poster Abstracts: Ke Zhang (Johns Hopkins University, USA)
"Ataxin-2 in Stress Granules Regulates Nucleocytoplasmic Transport in ALS"
9:25 pm - 9:30 pmDiscussion
Thursday
7:30 am - 8:30 amBreakfast
9:00 am - 12:30 pmAxons and Glial Cells
Discussion Leader: Richard Ransohoff (Biogen, USA)
9:00 am - 9:35 amBeth Stevens (Boston Children's Hospital, USA)
"How Microglia Prune Synapses in Health and Disease"
9:35 am - 9:45 amDiscussion
9:45 am - 10:20 amErika Holzbaur (University of Pennsylvania, USA)
"Autophagy Dynamics in Neuronal Homeostasis and Neurodegeneration"
10:20 am - 10:30 amDiscussion
10:30 am - 11:00 amCoffee Break
11:00 am - 11:35 amRichard Robitaille (University of Montreal, Canada)
"Glial Mismanagement of Neuromuscular Junction Structure and Function in ALS"
11:35 am - 11:45 amDiscussion
11:45 am - 11:55 amSelected 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 pmDiscussion
12:00 pm - 12:10 pmSelected 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 pmDiscussion
12:15 pm - 12:25 pmSelected from Poster Abstracts: Jonathan Grima (Johns Hopkins University, USA)
"The Nuclear Pore Complex Is Compromised in ALS"
12:25 pm - 12:30 pmDiscussion
12:30 pmLunch
1:30 pm - 4:00 pmFree Time
4:00 pm - 6:00 pmPoster Session
6:00 pmDinner
7:30 pm - 9:30 pmFrom Big Data Analysis to Therapeutics
Discussion Leader: Kenneth Fischbeck (National Institute of Neurological Disorders and Stroke, NIH, USA)
7:30 pm - 8:00 pmErnest 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 pmDiscussion
8:10 pm - 8:40 pmLeonard Petrucelli (Mayo Clinic College of Medicine and Science, USA)
"Mechanistic and Therapeutic Insights into C9ALS"
8:40 pm - 8:50 pmDiscussion
8:50 pm - 9:20 pmMatthew Disney (The Scripps Research Institute, USA)
"Design of Small Molecules Targeting Repeating Transcripts"
9:20 pm - 9:30 pmDiscussion
Friday
7:30 am - 8:30 amBreakfast
9:00 amDeparture
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