NPC1 KO mouse brain study

NPC1 KO mouse brain study

Well it has been a little while since I wrote a post, and a lot has happened since then, which I am sure you read about in our blogs and tweets. We changed our company name, partnered with Novartis and started partnering with patient groups to engineer our organisms...
Autophagic flux and NPC

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...
Phenotypic screens and target ID

Phenotypic screens and target ID

To continue on the theme of understanding your compound of interest’s mechanism of action, let’s discuss target identification (ID). You have just completed your screen and found a hit, but now you want to learn more about why it is a hit. As mentioned before,...
RNAseq and MOA

RNAseq and MOA

As the cell biology lead at Perlstein Lab, I spend a lot of time thinking about ways to better understand the mechanism of action of the hits from our primary screens. There are many experiments one can do to try to narrow down what pathway their compound is affecting...
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