Perlstein Lab

Leave no mutation behind

PLab is the first Public Benefit Corporation accelerating precision drug discovery for 5,000+ rare genetic diseases.

  • 1 in 12 people has an orphan disease. 95% of orphan diseases have no treatment.

  • Each disease is caused
    by a spectrum of genetic mutations.

  • Perlstein Lab studies each mutation
    in model organisms to produce precision medicine.


Autophagic flux and NPC

It has been a while since you have heard from me. We are deep in post screen characterization of our NPC hits so I thought I would take a step back to the beginning to discuss NPC disease biology. We focused a bit on the cholesterol and lipid metabolism aspects of Niemann Pick C disease […]