New Preprint Alert: Identification of compounds enhancing Aqp4 stop codon readthrough

Darshan led an elegant series of screens, with the goal to find drugs that might promote ‘glymphatic’ function, and perhaps slow disease. Starting with a series of reporters, a herculean screen of 2600 drugs, and counter screens, he finds an enhancer of this novel perivascular form of Aqp4 by altering stop codon readthrough. The final lead alters the endogenous protein in astrocytes. We’ve got a ways to go to show this can help in a disease directly, but it’s an important first lead for a preclinical tool. Aqp4 is needed for clearance of waste from brain, and this is the isoform that is positioned to mediate that. Finally, having something that regulates stop-codon readthrough gives us as a chance to understand the biology of this strange new phenomena as well. Congrats Darshan!