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Lysosomal Diseases (GRS)
Gordon Research Seminar

Decoding Lysosomal Signals to Understand Disease Mechanisms and Define New Therapeutic Strategies for Lysosomal Storage Disorders

Dates

March 4-5, 2017

Location

Renaissance Tuscany Il Ciocco
Lucca (Barga), Italy

Organizers

Chair:
Chiara Di Malta

Meeting 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 review innovative discoveries in the lysosomal diseases field. The meeting will cover different research areas spanning from basic lysosome biology to the pathogenetic mechanisms of lysosomal diseases and new therapeutic strategies for the treatment of these disorders.

Related Meeting

This GRS was held in conjunction with the "Lysosomal Diseases" Gordon Research Conference (GRC). Refer to the associated GRC program page for more information.

Contributors

Meeting Program

Saturday
1:30 pm - 4:30 pmArrival and Check-in
3:00 pm - 3:15 pmIntroductory Comments by GRC Site Staff / Welcome by the GRS Conference Chair
3:15 pm - 4:00 pmKeynote Session: Latest Understanding in the Biology of Lysosomal Storage Diseases
Discussion Leader: Gennaro Napolitano (TIGEM - Telethon Institute of Genetics and Medicine, Italy)
3:15 pm - 3:55 pmTimothy Cox (University of Cambridge, United Kingdom)
"Lysosomes: Coincidence, Convergence and Providence"
3:55 pm - 4:00 pmDiscussion
4:00 pm - 5:30 pmPoster Session
5:30 pm - 7:30 pmBasic Biology of Lysosomal Function
Discussion Leader: Doris Hoeglinger (University of Oxford, United Kingdom)
5:30 pm - 5:45 pmAnna Brown (Oregon State University, USA)
"Use of DSPE-PEG Micelles to Enable Efflux of Lipid Aggregates in Late Endosomes/Lysosomes"
5:45 pm - 5:50 pmDiscussion
5:50 pm - 6:05 pmPablo Contreras (Pontificia Universidad Catolica de Chile, Chile)
"c-Abl Mediates Tyrosine TFEB Phosphorylation and Its Cytoplasmic Localization: Implications in the NPC Cholesterol Lysosomal Storage Disease"
6:05 pm - 6:10 pmDiscussion
6:10 pm - 6:25 pmSaskia Heybrock (Christian-Albrechts-University Kiel, Germany)
"Role of the Lysosomal Integral Membrane Protein Type 2 in Lysosomal Cholesterol Transport"
6:25 pm - 6:30 pmDiscussion
6:30 pm - 6:45 pmYang Li (Institute of Genetics and Developmental Biology, Chinese Academy of Sciences, China)
"Natural Compounds as Probes to Dissect Lysosome Homeostasis"
6:45 pm - 6:50 pmDiscussion
6:50 pm - 7:05 pmLeopoldo Staiano (Telethon Institute of Genetics and Medicine (TIGEM), Italy)
"OCRL Controls a Lysosomal Response Triggered by Autophagosome-Lysosome Fusion"
7:05 pm - 7:10 pmDiscussion
7:10 pm - 7:25 pmRenata Voltolini Velho (University Medical Center Hamburg-Eppendorf, Germany)
"Regulation of Site-1 Protease-Mediated Cleavage of the GlcNAc-1-Phosphotransferase"
7:25 pm - 7:30 pmDiscussion
8:00 pmDinner
Sunday
7:30 am - 8:30 amBreakfast
9:00 am - 11:00 amDiseases Mechanisms and Therapeutic Strategies in Lysosomal Storage Disorders
Discussion Leader: Luis Tecedor (Children's Hospital of Philadelphia, USA)
9:00 am - 9:15 amRosa Bartolomeo (Telethon Institute of Genetics and Medicine (TIGEM), Italy)
"Targeting mTORC1 and Autophagy as New Therapy for Lysosomal Storage Disorders"
9:15 am - 9:20 amDiscussion
9:20 am - 9:35 amAlana Gibson (National Human Genome Research Institute, NIH, USA)
"Systemic AAV9 Gene Therapy Improves the Lifespan of Mice with Niemann-Pick Disease, Type C1"
9:35 am - 9:40 amDiscussion
9:40 am - 9:55 amHelene Gleitz (University of Manchester, United Kingdom)
"Whole Body Correction of Severe Mucopolysaccharidosis Type II by Lentiviral-Mediated Stem Cell Gene Therapy with Blood-Brain Barrier-Crossing Peptides"
9:55 am - 10:00 amDiscussion
10:00 am - 10:15 amNatalia Gomez-Ospina (Stanford University, USA)
"Correction of Mucopolysaccharidosis Type 1: Targeting Safe Harbor Loci Using Genome Editing"
10:15 am - 10:20 amDiscussion
10:20 am - 10:35 amJames Gray (University of Oxford, United Kingdom)
"Mechanisms of Remyelination Following Heat Shock Protein Treatment in Niemann-Pick Type C Mouse"
10:35 am - 10:40 amDiscussion
10:40 am - 10:55 amJlenia Monfregola (Telethon Institute of Genetics and Medicine (TIGEM), Italy)
"Generation of a Lysosomal Storage Disorder CRISPr Biobank for the Study of Lysosomal Storage Disorders"
10:55 am - 11:00 amDiscussion
11:00 am - 12:30 pmPoster Session

Coffee will be served in the poster area from 11:00 am - 11:30 am
12:30 pmLunch
1:30 pm - 2:30 pmMentorship Component: Career Mentoring Session
Discussion Leaders: Stephanie Cologna (University of Illinois at Chicago, USA) and Mylene Huebecker (University of Oxford, United Kingdom)
1:30 pm - 2:30 pmPanel Discussion
"What Options Do I Have for My Scientific Career?"
  • Thomas Braulke (University Medical Center Hamburg-Eppendorf, Germany)
  • Nicola Brunetti-Pierri (Telethon Institute of Genetics and Medicine (TIGEM), Italy)
  • Graciana Diez Roux (Telethon Institute of Genetics and Medicine (TIGEM), Italy)
  • Susan Kalled (Shire, USA)
2:30 pm - 3:00 pmEvaluation Period
Fill in GRS Evaluation Forms
3:00 pmSeminar Concludes

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 R13NS100248. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.
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