by Ethan Perlstein | Apr 10, 2016 | Blogposts, Science |
Five months ago we introduced PERL101, one of our lead compounds for Niemann-Pick Type C (NPC) disease, in Part One. Here in Part Two we pick up the thread. On this journey we’ve progressed from nematodes to patient cells to mice with an unoptimized primary...
by Tom Hartl | Sep 7, 2015 | Science |
Our medicinal chemistry advisor and pharm/tox consultant have asked me, “Do flies have livers?” I didn’t fully appreciate why they were asking me this until learning more about drug metabolism. In the body, foreign substances, or “xenobiotics,” (pesticides, cleaners,...
by Sangeetha Iyer | Aug 25, 2015 | Science |
Now that we’ve completed our screen in all of our invertebrate models of Niemann Pick Type C (NPC) disease, it’s time for us to switch gears to the higher models of NPC. We are in the process of moving our validated screen hits for additional testing in a...
by Nina DiPrimio | Jul 29, 2015 | Science |
For this post we are going to take a slight detour and talk about something related to Niemann-Pick C, filovirus infection. I am personally interested in the topic because of my love of viruses – plug for the highly informative TWiV (This Week in Virology)...
by Tom Hartl | Jul 15, 2015 | Science |
Niemann-Pick C (NPC) is a tragic neurodegenerative disease that is often referred to as childhood Alzheimer’s disease. 95% of patients with NPC carry mutations in the npc1 gene, which codes for the Npc1 protein. Npc1 resides in lysosomes and is necessary for...
by Sangeetha Iyer | Jul 7, 2015 | Science |
This recap is a tad bit late- I’ve been meaning to write about my last conference visit. I was at the 20th International C. elegans meeting held at UCLA from June 24th-28th 2015. Below are the highlights of the meeting. Disclaimer: This post is not meant to...
