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Lysosomal Diseases (GRS)

Gordon Research Seminar

Mechanisms of Pathogenesis and Therapeutic Challenges in Lysosomal Diseases

May 13 - 14, 2023

Chairs

and

Rey Don Jaime Grand Hotel

Avinguda de l'Hotel

Castelldefels, Barcelona, Spain

Venue and Travel Information

Conference Description

The Lysosomal Diseases GRS provides a unique forum for young doctoral and post-doctoral researchers to present their work, discuss new methods, cutting edge ideas, and pre-published data, as well as to build collaborative relationships with their peers. Experienced mentors and trainee moderators will facilitate active participation in scientific discussion to allow all attendees to be engaged participants rather than spectators.

The Seminar will explore the latest advancement in the field, focusing on introducing early career scientists to Lysosomal Diseases in a manner that engages interdisciplinary research. Attendees will have the chance to present their novel data on basic biology of the lysosome, novel tools to understand lysosome function, pathophysiology of diseases, innovative screening methods and therapeutic strategies.

Related Meeting

This GRS will be held in conjunction with the "Lysosomal Diseases" Gordon Research Conference (GRC). Those interested in attending both meetings must submit an application for the GRC in addition to an application for the GRS. Refer to the associated GRC program page for more information.

Conference Program

Saturday
1:00 pm - 5:00 pm	Arrival and Check-in
3:00 pm - 3:15 pm	Introductory Comments by GRC Staff / Welcome and Introduction from the Chairs
3:15 pm - 4:00 pm	Keynote Session: A Personal Journey Through Lysosomal Diseases - Career in Science and How to Become a Successful Researcher Discussion Leader:
3:15 pm - 3:20 pm	Introduction by Discussion Leader
3:20 pm - 3:55 pm	"From Caterpillars to Lysosomal Diseases, a Scientific Journey"
3:55 pm - 4:00 pm	Discussion
4:00 pm - 5:30 pm	Poster Session
5:30 pm - 7:30 pm	Pathological Mechanisms of Lysosomal Diseases Discussion Leader:
5:30 pm - 5:45 pm	"Unveiling the Molecular Mechanisms of Batten Disease in the Absence of CLN3"
5:45 pm - 5:50 pm	Discussion
5:50 pm - 6:05 pm	"The Role of the NPC Pathway in Salmonella Infection"
6:05 pm - 6:10 pm	Discussion
6:10 pm - 6:25 pm	"Molecular Mechanisms of Increased Gout in Patients With Clonal Hematopoiesis by DNMT3A Mutations and Gout"
6:25 pm - 6:30 pm	Discussion
6:30 pm - 6:45 pm	"Neu1 Modulates Trem2 Signaling and Microglial Function"
6:45 pm - 6:50 pm	Discussion
6:50 pm - 7:05 pm	"Progranulin and Prosaposin Require LRP10, a Parkinson’s Disease Gene, for Their Efficient Trafficking to Lysosomes"
7:05 pm - 7:10 pm	Discussion
7:10 pm - 7:25 pm	"Multiomic Analysis of iPSC-derived Neuronal Model from Sib Pairs With Gaucher Disease Discordant for Parkinson Disease"
7:25 pm - 7:30 pm	Discussion
8:00 pm - 9:00 pm	Dinner
Sunday
7:30 am - 8:30 am	Breakfast
9:00 am - 11:00 am	Novel Models and Therapeutic Strategies for Lysosomal Diseases Discussion Leader:
9:00 am - 9:15 am	"From Mouse to Sheep: Generating a Sheep Model and Developing a Gene Therapy for Sialidosis"
9:15 am - 9:20 am	Discussion
9:20 am - 9:35 am	"Assessing the Effect of Dosage and Injection Route on AAV Gene Therapy in The Treatment of Feline GM2 Gangliosidosis"
9:35 am - 9:40 am	Discussion
9:40 am - 9:55 am	"Developing New Models to Test Proteostatic Therapies for Niemann-Pick type C Disease"
9:55 am - 10:00 am	Discussion
10:00 am - 10:15 am	"Investigating the Ability of Granulin Subunits to Rescue Lysosome Dysfunction, Inflammation, and Disease-like Pathology in a Mouse Model of Progranulin Deficiency"
10:15 am - 10:20 am	Discussion
10:20 am - 10:35 am	"Humanized I1061T-NPC1 Mice as a New Model of Niemann-Pick Type C Disease"
10:35 am - 10:40 am	Discussion
10:40 am - 10:55 am	"Elucidating Expression of a Corrective Enzyme for MPS I Through Administration of an Adenoviral Vector Targeted to Endothelial Cells"
10:55 am - 11:00 am	Discussion
11:00 am - 12:30 pm	Poster Session Coffee will be served in the poster area from 11:00 am - 11:30 am
12:30 pm - 1:30 pm	Lunch
1:30 pm - 2:30 pm	Mentorship Component: Different Career Paths in Science: Prospectives from Academia and Industry Discussion Leader:
1:30 pm - 2:30 pm	Panel Discussion Science Work Experiences in Academia and Industry
2:30 pm - 3:00 pm	Evaluation Period Complete the GRS Evaluation Forms; Election of Future Chair(s)
3:00 pm	Seminar Concludes

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Conference History Lysosomal Diseases

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Lysosomal Diseases

March 2 - 7, 2025

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