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  DAY 1: Tuesday, May 4, 2004


  8:00 AM Registration and Continental Breakfast

  8:30 AM

Opening Remarks
Allen Spiegel, MD
Director, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health

Steve Groft, PharmD

Director, Office of Rare Diseases, National Institutes of Health

  8:40 AM

Introductory Address
Peter Walter, PhD, University of California at San Francisco
Signaling from the ER to the Nucleus


  9:15 AM SESSION I

The ER as a Site for Protein Folding, Complex Assembly, and Quality Control for the Secretory Pathway
Chair: Dieter H. Wolf, PhD, Universitaet Stuttgart

Hidde Ploegh, PhD, Harvard University
TBD

Charlie Barlowe, PhD, Dartmouth University
TBD

Dieter H. Wolf, PhD, Universitaet Stuttgart
Protein Quality Control in the Endoplasmic Reticulum and the Ubiquitin-proteasome-chaperone Connection

Randy Hampton, PhD, University of California, San Diego
Reversible, Small Molecule Regulation of ER Quality Control


  10:45 AM Break

  11:00 AM SESSION II

The Unfolded Protein Response: Maintenance of Proper Folding in the ER
Chair: David Ron, MD, New York University School of Medicine

Doug Cavener, PhD, Pennsylvania State University
PERKs and the Unfolded Protein Response

Linda Hendershot, PhD, St. Jude Children's Research Hospital
ER Complexes and BIP Regulators

David Ron, MD, New York University School of Medicine
Translational Control in the Unfolded Protein Response

Jeff Brodsky, PhD, University of Pittsburgh
Molecular Chaperone Action and Cellular Responses During ERAD


  12:30 PM Lunch

  2:00 PM Keynote Lecture
William E. Balch, PhD, The Scripps Research Institute
Protein Traffic in Conformational Diseases

  2:45 PM SESSION III

ER Stress and Mis-folding

Gert Kreibich, PhD, New York University School of Medicine
Membrane Protein Assembly and Targeting in the Bladder Epithelium

Craig B. Thompson, PhD, University of Pennsylvania
Programmed Cell Death Induced by ER Stress

Sanjay Nigam, MD, University of California, San Diego

Invited short presentations selected from abstracts


  5:30 PM Poster Session/Reception





  DAY 2: Wednesday, May 5, 2004


  7:30 AM Continental Breakfast

  8:00 AM Introductory Address
Jeffrey W. Kelly, PhD, The Scripps Research Institute
An Overview of Misfolding Diseases and Therapeutic Approaches


  8:35 AM SESSION IV

Defective Protein Folding and Targeting in Renal Disease
Chair: Jeffrey W. Kelly, PhD, The Scripps Research Institute

Michel Bouvier, PhD, Universite de Montreal
Pharmacological Chaperones; A Potential Therapeutic Strategy for Nephrogenic Diabetes Insipidus and Other Conformational Diseases

Chris Danpure, PhD, University College London
Primary Hyperoxaluria

Peter Agre, MD, PhD, Johns Hopkins University
Aquaporins

Karl Tryggvason, MD, PhD, University of Oulu
Folding and Nephrin


  10:05 AM Break

  10:20 AM SESSION V

Mis-folding and Disease
Chair: David H. Perlmutter, MD, University of Pittsburgh School of Medicine

David H. Perlmutter, MD, University of Pittsburgh School of Medicine
Alpha 1 Antitrypsin Deficiency: Retention of an Aggregated Protein in ER Causes Liver Disease

David Ellison, MD, Oregon Health & Science University
Misfolding and ER Retention of Renal NCC in Gitelman’s Syndrome

Show-Ling Shyng, PhD, Oregon Health & Science University
Defective Trafficking of K ATP Channels in Congenital Hyperinsulinism

Invited short presentation selected from abstracts


  11:50 AM Lunch

  1:00 PM SESSION VI

Mis-folding and Disease
Chair: David H. Perlmutter, MD, University of Pittsburgh School of Medicine

Jean Baum, PhD, Rutgers University
NMR and Folding Disease

Dr. Laura S. Itzhaki, Hutchison/MRC Research Centre
The Role of Domain Swapping in Protein Aggregation

Mark McNiven, PhD, Mayo Clinic
Dynamin and Endocytosis in Hepatocytes


 

  2:10 PM Break

  2:25 PM SESSION VII

Mis-folding, Disease and Therapeutic Approaches
Chair: Pam Zeitlin, MD, PhD, Johns Hopkins University

Alan Verkman, MD, PhD, University of California at San Francisco
Small Molecule Therapy and CF

P. Michael Conn, PhD, Oregon Health & Science University
Origami of the GnRH Receptor – A New Therapeutic Approach Unfolding

Pam Zeitlin MD, PhD, Johns Hopkins School of Medicine
Pharmacologic Approaches to CFTR Misfolding

Bob Desnick, MD, PhD, Mount Sinai School of Medicine
Chaperone-mediated Enzyme Enhancement Therapy


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National Institute of Diabetes and Digestive and Kidney Diseases