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Lysosomal Diseases (GRS)

Gordon Research Seminar

Lysosomal Pathogenesis and Its Translation to New Treatment Strategies for Lysosomal Disorders

March 2 - 3, 2019
Chairs and
Grand Galvez
2024 Seawall Boulevard
Galveston, Texas, United States
 Venue and Travel Information

Conference Description

The Gordon Research Seminar on Lysosomal Diseases is a unique forum for graduate students, post-docs, and other scientists with comparable levels of experience and education to present and exchange new data and cutting edge ideas.

The focus of this meeting is to introduce young researchers to emerging advances in the lysosomal disease field. This meeting will cover a broad spectrum of research, from relevant discoveries in lysosomal basic biology and lysosomal pathogenesis to their translation to new treatment strategies for lysosomal disorders.

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
2:00 pm - 5:00 pm
Arrival and Check-in
3:30 pm - 3:45 pm
Introductory Comments by GRC Site Staff / Welcome from the GRS Chair
3:45 pm - 4:30 pm
Keynote Session: Developing Therapeutical Strategies for Lysosomal Storage Diseases
Discussion Leader:
3:45 pm - 4:20 pm
"Unexpected Adventures in Lysosomal Disease Research"
4:20 pm - 4:30 pm
Discussion
4:30 pm - 6:00 pm
Poster Session
6:00 pm - 7:00 pm
Dinner
7:30 pm - 9:30 pm
Lysosomal Pathology: New Models, New Approaches
Discussion Leader:
7:30 pm - 7:45 pm
"Neuronal Network Dysfunction in Juvenile Neuronal Ceroid Lipofuscinosis"
7:45 pm - 7:50 pm
Discussion
7:50 pm - 8:05 pm
"Modelling the Role of Lysosome Dysfunction in the Retinal Pigment Epithelium in the Development of Early Age-Related Macular Disease"
8:05 pm - 8:10 pm
Discussion
8:10 pm - 8:25 pm
"Using Drosophila melanogaster to Uncover Disease-Related Mechanisms of Sanfilippo Syndrome"
8:25 pm - 8:30 pm
Discussion
8:30 pm - 8:45 pm
"Cell-Autonomous Expression of the Lysosomal Hydrolase GALC: A New Model of Krabbe Disease"
8:45 pm - 8:50 pm
Discussion
8:50 pm - 9:05 pm
"Early Spinal Cord Vulnerability in CLN1 Disease Reveals an Important and Novel Therapeutic Target"
9:05 pm - 9:10 pm
Discussion
9:10 pm - 9:25 pm
"Broken Hearts in Juvenile Dementia: Investigating Cardiac Myocytes in CLN3 Disease"
9:25 pm - 9:30 pm
Discussion
Sunday
7:30 am - 8:30 am
Breakfast
9:00 am - 11:00 am
Therapeutic Strategies in Lysosomal Storage Disorders
Discussion Leader:
9:00 am - 9:15 am
"Cannabidiol for the Treatment of Seizures in CLN1 Disease"
9:15 am - 9:20 am
Discussion
9:20 am - 9:35 am
"Acetyl Leucine as Novel Therapy for Lysosomal Storage Diseases"
9:35 am - 9:40 am
Discussion
9:40 am - 9:55 am
"Enzyme Replacement Therapy with Recombinant Pro-Cathepsin-D Corrects Defective Proteolysis and Autophagy in Neuronal Ceroid Lipofuscinosis"
9:55 am - 10:00 am
Discussion
10:00 am - 10:15 am
"Proteomic Analysis of Brain and Liver Tissues Reveals Altered Proteins in the Symptomatic Niemann-Pick, Type C1 Mouse Model"
10:15 am - 10:20 am
Discussion
10:20 am - 10:35 am
"Identifying a Biomarker Signature for Batten Disease"
10:35 am - 10:40 am
Discussion
10:40 am - 10:55 am
"Deep Transcriptomic Analysis of Pompe Mice Treated with Liver Expression of Secretable GAA Reveals Significance of Bioenergetic Homeostasis"
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: Career Mentoring: Different Paths on the Science Track
Discussion Leader:
1:30 pm - 2:30 pm
Panel Discussion
Different Paths on the Science Track
2:30 pm - 3:00 pm
Evaluation Period
Fill in GRS Evaluation Forms
3:00 pm
Seminar Concludes

Contributors

National Institutes of Health
Voyager Therapeutics, Inc.

Gauchers Association - Susan Lewis Memorial Award

Hide and Seek Foundation
The Children
Niemann-Pick UK (NPUK)

Research reported in this publication was supported by the National Institute Of Neurological Disorders And Stroke of the National Institutes of Health under Award Number R13NS111940. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.

Conference Links

Conference History Lysosomal Diseases
Additional Fee Info



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