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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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