Protein Misfolding and Rare Genetic Diseases:
From Human Genetics to Drug Discovery

New England Structural Biology Association (NESBA) presents a one day conference on Protein Misfolding and Rare Genetic Diseases: From Human Genetics to Drug Discovery

LaCava Center
Bentley University
Waltham, MA 
Friday, March 29, 2013 
8:00 am - 6:00 pm

Loss of protein homeostasis (proteostasis) underlies numerous disease states from age-related diseases like Alzheimer's to genetic disorders such as cystic fibrosis. This one-day conference will highlight cellular mechanisms required to maintain proteostasis - from ensuring a properly folded structure to managing misfolded or aggregated proteins - and present new ideas and pharmacological tools to repair loss of proteostasis.

Schedule:

Morning Session: Molecular Mechanisms and Quality Control of Protein Folding
Afternoon Session: Misfolding and Aggregation Diseases and Small Molecule Therapies

8:00am-8:45am Registration and light breakfast
8:45am-9:00am Opening Remarks

The Proteostasis Network
9:00am-9:45am Peter Reinhart, Proteostasis Therapeutics Inc.
Developing proteostasis network regulators as novel therapeutics

Molecular Mechanisms and Quality Control of Protein Folding
10:00am-10:45am Lila Gierasch, University of Massachusetts, Amherst
Allosteric mechanism of Hsp70 molecular chaperones
11:00am-11:15am Coffee Break and Networking
11:15am-12:00pm Jeff Brodsky, University of Pittsburgh
Targeting misfolded proteins for degradation
12:15pm-1:15pm  Lunch

Misfolding and Aggregation Diseases and Small Molecule Therapies
1:15pm-2:00pm Paul Negulescu, Vertex Pharmaceuticals
Can we fix the broken protein that causes Cystic Fibrosis?
2:15pm-2:35pm Jared Auclair, Brandeis University 
Covalent interactions as a therapeutic approach for stabilizing superoxide dismustase (SOD1), the protein destabilized in the most common form of familial amyotrophic lateral sclerosis
2:40pm-3:25pm
David Perlmutter, University of Pittsburgh
Novel treatment strategies for alpha1-antitrypsin deficiency by targeting endogenous proteostasis
3:40pm-4:00pm Coffee Break and networking
4:00pm-4:45pm Gregory Petsko, Brandeis University and Weill Cornell Medical College
Pharmacological chaperones for the retromer protein complex: A new therapeutic approach for Alzheimer's and Parkinson's Disease
5:00pm-5:45pm Daniel Finley, Harvard Medical School
Enhancement of proteasome activity by a small-molecule inhibitor of USP14
6:00pm-7:00pm Closing remarks, Mixer, and Networking

Meeting Organizers:

Meeting Sponsors:

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