Derek Lowe's commentary on drug discovery and the pharma industry. An editorially independent blog from the publishers of Science Translational Medicine. All content is Derek’s own, and he does not in any way speak for his employer.
As many will know, there is a whole set of what are termed “polyglutamine repeat disorders“, which themselves are a large part of a bigger set of trinucleotide repeat disorders. Huntington’s disease is a well-known one: the gene (HTT) for the huntingtin protein ends up with a series of CAG nucleotide repeats, which after translati… Read More
OK, let’s get reductionist, and let’s see why getting reductionist often works so well. How do you know when your finger has touched something? You feel it – but how do you feel it? Your nerves have sent an impulse to your brain, which interprets it as something having physically come into contact with your… Read More
Well, it’s inadvertently been sort of a Neuroscience Week here. This latest paper is a very interesting addition to the field indeed, just out from a very large team centered at the Allen Institute, where some rather large-scale work in the field has been done in the past. This one continues their tradition: it’s a… Read More
When you look closely at cellular biochemistry, what you see are a lot of amazing processes that are surrounded by amazing amounts of redundancy, backups, patches, and accumulated tweaks and fixes. That’s evolution for you; these things have been piling up for a billion years or two, and we’re all the descendants of the critters… Read More
I’ve written several times here about phase-separated condensates in cells, but now comes a rarity: a paper with some evidence for a therapeutic application. Everyone in the field has been thinking along such lines, naturally, but this is the first small-molecule screen that I’ve seen that tries to tie modifying condensate behavior in t… Read More
Here’s a new look at the various ways that small molecules can affect a well-known drug target (the estrogen receptor) and it shows us that we’re all going to have to look at these things more carefully than we do. Now, to be fair, the ER is already fairly complicated, because it’s a nuclear receptor. Read More
This is a pretty interesting paper on several levels. It sheds light on Mucin I kidney disease (MKD), on protein degradation pathways (a hot topic these days, as those in the industry well know), and it also provides a small molecule lead compound. It’s a large multicenter team, starting off with the Broad Institute, but… Read More
I wrote here about a new company (Homology Medicine) that claimed to have a viral method for gene editing that did not involve any sort of double-strand DNA breaking enzyme (as you need during the CRISPR, TALENs, or zinc-finger nuclease methods). That’s a pretty interesting claim, because double-strand breaks (DSBs) are powerful but can be… Read More
I’ve written here about phase-separated condensates inside cells, and the publications on these continue to show up all over the literature. I found this recent one in Science to be particularly interesting, on several levels. One thing the condensate idea has given a framework to is the variety of small cellular structures that have been… Read More
The Wall Street Journal published a provocative article the other day, entitled “Don’t Understand Moronic Bromides?” about the proliferation over the years of acronyms in science.(Note the old-fashioned usage of “bromide” derived from the early sleeping pills). And while it’s a cranky piece, it’s not wrong. Read More
