Perlstein Lab Overview
Dr. Ethan Perlstein
Wednesday, March 2, 2016
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Chapters
Introduction
Summary of PLab PBC
Core Team
Board of Directors
Orphan diseases
We need Moore’s Law for orphan diseases
PLab platform overview
Rare disease genes are ancient
Spectrum of mutations
Scalable CRISPR-enabled platform
Reversing disease in whole animals
Enabling smart small n trials
Picture 1.
5 reasons to use model orgs in drug discovery
PLab is initially targeting childhood monogenic diseases
NPC1 is required for cholesterol trafficking
In absence of NPC1
NPC fly model and screen
NPC worm model and screen
NPC patient fibroblast model
PLab's Niemann-Pick Type C program
Chemotypes alter cholesterol distribution
All chemotypes induce “bypass” vesicles
PERL101 redistributes cholesterol in the cell
Submicromolar analogs in PERL101 series
PERL 101 treated cells
Secondary endpoint in worms:
Secondary endpoint in worms: Maturation
Mouse liver microsome stability
PERL101 series mouse PK (IV & oral)
Summary and next steps
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