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Amyotrophic Lateral Sclerosis (ALS) and Related Motor Neuron Diseases

Gordon Research Conference

Mechanisms, Modeling and Translational Therapeutics

July 2 - 7, 2023

Chairs

and

Vice Chairs

and

Les Diablerets Conference Center

Eurotel Victoria

Les Diablerets, Vaud (fr), Switzerland

Venue and Travel Information

Conference Description

The Amyotrophic Lateral Sclerosis (ALS) and Related Motor Neuron Diseases GRC is a premier, international scientific conference focused on advancing the frontiers of science through the presentation of cutting-edge and unpublished research, prioritizing time for discussion after each talk and fostering informal interactions among scientists of all career stages. The conference program includes a diverse range of speakers and discussion leaders from institutions and organizations worldwide, concentrating on the latest developments in the field. The conference is five days long and held in a remote location to increase the sense of camaraderie and create scientific communities, with lasting collaborations and friendships. In addition to premier talks, the conference has designated time for poster sessions from individuals of all career stages, and afternoon free time and communal meals allow for informal networking opportunities with leaders in the field.

The 2023 Gordon Conference brings together an exciting agenda of topics that highlights recent scientific and clinical advances for ALS, ALS/FTD and related motor neuron diseases. The conference will feature a wide range of topics that focus on disease basic biology and mechanism, such as TDP-43 based neurodegeneration and cryptic splicing, neuroinflammation and vascular biology, axonal biology, as well as genetic modifiers of motor neuron disease. In addition to the mechanistic science, there will be an emphasis on translational science, involving preclinical animal models, therapeutic strategies, biomarkers, and the review of recent clinical trials.

Related Meeting

This GRC will be held in conjunction with the "Amyotrophic Lateral Sclerosis (ALS) and Related Motor Neuron Diseases" Gordon Research Seminar (GRS). Those interested in attending both meetings must submit an application for the GRS in addition to an application for the GRC. Refer to the associated GRS program page for more information.

Conference Program

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: Translational Science and Therapeutic Landscape Discussion Leaders: and
7:40 pm - 8:00 pm	Introduction by Discussion Leader
8:00 pm - 8:45 pm	"Regulated Multi-Phase Transitions: TDP-43, Anisosomes, Aggregation, and Neurodegeneration"
8:45 pm - 9:10 pm	Discussion
9:10 pm - 9:30 pm	General Discussion
Monday
7:30 am - 8:30 am	Breakfast
9:00 am - 12:30 pm	TDP-43 and Neurodegeneration: Loss of Function, Cryptic Splicing, and Therapeutics Discussion Leader:
9:00 am - 9:15 am	Introduction by Discussion Leader
9:15 am - 9:35 am	"When, Where and How to Stop TDP-43 Cryptic Splicing"
9:35 am - 9:45 am	Discussion
9:45 am - 10:05 am	"Pathogenic Mechanism and Therapeutic Potential for Misfolded TDP-43 Oligomers in ALS"
10:05 am - 10:15 am	Discussion
10:15 am - 10:40 am	Coffee Break
10:40 am - 11:00 am	"RNA Binding Proteins Aggregation in ALS"
11:00 am - 11:10 am	Discussion
11:10 am - 11:30 am	"Structural Insights into TDP43 Mislocalization in ALS/FTD Models"
11:30 am - 11:40 am	Discussion
11:40 am - 12:00 pm	"TDP-43 Splicing Biology and Cryptic Peptides as Potential Biomarkers"
12:00 pm - 12:10 pm	Discussion
12:10 pm - 12:30 pm	General Discussion
12:30 pm - 1:30 pm	Group Photo / 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:
4:30 pm - 6:00 pm	Poster Session
6:00 pm - 8:00 pm	Emerging Genetic and Mechanistic Modifiers of ALS and FTD Discussion Leader:
6:00 pm - 6:20 pm	Introduction by Discussion Leader
6:20 pm - 6:40 pm	"Structures of Pathological TDP-43 Filaments in ALS and FTLD"
6:40 pm - 6:50 pm	Discussion
6:50 pm - 7:10 pm	"Molecular Mechanisms of RNA Binding Protein Dysfunction in ALS and Related Neurodegenerative Disorders"
7:10 pm - 7:20 pm	Discussion
7:20 pm - 7:40 pm	"Binding to RNA and Protein Assembly Regulate TDP-43 Homeostasis and Cellular Dynamics"
7:40 pm - 7:50 pm	Discussion
7:50 pm - 8:00 pm	General Discussion
8:00 pm - 9:00 pm	Dinner
Tuesday
7:30 am - 8:30 am	Breakfast
9:00 am - 12:30 pm	Axonal and Synapse Biology; Degeneration in Motor Neuron Diseases Discussion Leader:
9:00 am - 9:20 am	Introduction by Discussion Leader
9:20 am - 9:40 am	"Motor Proteins in Motor Neuron Diseases; Can We Get There in Time?"
9:40 am - 9:50 am	Discussion
9:50 am - 10:10 am	"tRNA Synthetase-Associated Peripheral Neuropathies: Mechanisms and Therapeutic Strategies"
10:10 am - 10:20 am	Discussion
10:20 am - 10:45 am	Coffee Break
10:45 am - 11:05 am	"Is ALS a Distal Disease? The Interplay Between Local Synthesis and Neuromuscular Degeneration"
11:05 am - 11:15 am	Discussion
11:15 am - 11:35 am	"Uncovering Mechanisms of Axon Maintenance and Demise in ALS"
11:35 am - 11:45 am	Discussion
11:45 am - 11:55 am	"Scalable, Optically-Active Human Neuromuscular Junction Model Reveals Convergent Mechanisms of Synaptic Dysfunction in Familial ALS."
11:55 am - 12:00 pm	Discussion
12:00 pm - 12:10 pm	"Axonal Stress Triggers a Transport-Independent Somatic Stress Response"
12:10 pm - 12:15 pm	Discussion
12:15 pm - 12:25 pm	"Identification of a New Functional Interaction Between TDP-43 and USP10"
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	Neuroinflammation and Vascular Biology in ALS and FTD Discussion Leader:
6:00 pm - 6:20 pm	Introduction by Discussion Leader
6:20 pm - 6:40 pm	"Neuroimmune Dysfunction in FTD-ALS Spectrum Disorders"
6:40 pm - 6:50 pm	Discussion
6:50 pm - 7:10 pm	"Impact of C9orf72 Mutations in the Biology of Microglia"
7:10 pm - 7:20 pm	Discussion
7:20 pm - 7:45 pm	"Biogen C9ORF72 ASO data - Antisense Oligonucleotide Therapy for C9orf72 ALS: Unexpected Clinical and Biological Results from the Biogen/Ionis BIIB078 Phase 1 Clinical Trial. Should Antisense Targeting C9orf72 Now be Investigated?"
7:45 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	Astrocyte Biology in ALS and FTD Discussion Leader:
9:00 am - 9:05 am	Introduction by Discussion Leader
9:05 am - 9:25 am	"A New Role for an Archaic Model: Release of Inorganic Polyphosphate byALS/FTD Astrocytes Causes Non-Cell-Autonomous Toxicity to Motoneurons"
9:25 am - 9:35 am	Discussion
9:35 am - 9:55 am	"The Involvement of Glia in Frontotemporal Dementia"
9:55 am - 10:05 am	Discussion
10:05 am - 10:30 am	Coffee Break
10:30 am - 10:50 am	"Glial Signaling and Neurodegeneration"
10:50 am - 11:00 am	Discussion
11:00 am - 11:20 am	"Astrocytes for Neuroprotection"
11:20 am - 11:30 am	Discussion
11:30 am - 11:50 am	"Aberrant Activation of CHMP7/ESCRT-III Nuclear Pore Complex Surveillance as an Initiator of Pathophysiological Cascades in ALS/FTD"
11:50 am - 12:00 pm	Discussion
12:00 pm - 12:10 pm	"Detection of Cryptic Exons in the Frontal Cortex of C9orf72-FTD Patients via Single Nuclei RNA Sequencing"
12:10 pm - 12:15 pm	Discussion
12:15 pm - 12:25 pm	"Single-Cell Integrated RNA-Sequencing and ATAC-Sequencing in Human Motor Cortex Identifies Cell-Specific Genetic Drivers of ALS"
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	Translational Biomarkers Discussion Leader:
6:00 pm - 6:15 pm	Introduction by Discussion Leader
6:15 pm - 6:35 pm	"Species Differences in Interpretation of the TSPO PET Signal"
6:35 pm - 6:40 pm	Discussion
6:40 pm - 7:00 pm	"Progressive Motor and Extra-Motor Cerebral Degeneration in ALS"
7:00 pm - 7:05 pm	Discussion
7:05 pm - 7:25 pm	"Emerging Pathological Mechanism and Biomarkers in Amyotrophic Lateral Sclerosis"
7:25 pm - 7:30 pm	Discussion
7:30 pm - 7:40 pm	"NEK1 Loss-of-Function Mutation Impairs Ciliogenesis in iPSC-Motoneurons"
7:40 pm - 7:45 pm	Discussion
7:45 pm - 7:55 pm	"TDP-43 SUMOylation is a Stress Responsive Modification Linked to Neurodegeneration"
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	Translational Science in ALS and FTD Discussion Leader:
9:00 am - 9:05 am	Introduction by Discussion Leader
9:05 am - 9:25 am	"RNA-Targeted Therapies for ALS"
9:25 am - 9:35 am	Discussion
9:35 am - 9:55 am	"Silencing FUS and Other Rare Genetic Forms of ALS"
9:55 am - 10:05 am	Discussion
10:05 am - 10:30 am	Coffee Break
10:30 am - 10:50 am	"Antisense Oligonucleotides (ASOs) for the Treatment of Amyotrophic Lateral Sclerosis (ALS)"
10:50 am - 11:00 am	Discussion
11:00 am - 11:20 am	"Standardized Translational Approach to Target Motor and Sensory Neurons Via Intrathecal Administration of AAV9"
11:20 am - 11:30 am	Discussion
11:30 am - 11:50 am	"Lessons Learned from Treating Spinal Muscular Atrophy"
11:50 am - 12:00 pm	Discussion
12:00 pm - 12:10 pm	"Combined Rescue of STMN2 & UNC13A Mis-Splicing in ALS Using A Novel U7 snRNA Therapeutic Approach"
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 - 4:30 pm	Free Time
4:30 pm - 6:00 pm	Poster Session
6:00 pm - 8:00 pm	Late-Breaking Topics Discussion Leader:
6:00 pm - 6:20 pm	"TDP-43 in ALS"
6:20 pm - 6:30 pm	Discussion
6:30 pm - 6:50 pm	"New Epigenetic Awareness at an ALS Locus"
6:50 pm - 7:00 pm	Discussion
7:00 pm - 7:20 pm	"C9orf72 Acts in Myeloid and Lymphoid Lineages to Regulate The IL-17 Inflammatory Axis"
7:20 pm - 7:30 pm	Discussion
7:30 pm - 7:50 pm	"Two New ALS Drug Targets (And One Drug)"
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

Contributors

Conference Links

Conference History Amyotrophic Lateral Sclerosis (ALS) and Related Motor Neuron Diseases (GRS)

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