Peer-Reviewed Publication COLD SPRING HARBOR LABORATORY Spinal muscular atrophy, or SMA, is the leading genetic cause of infant death. Less than a decade ago, Cold Spring Harbor Laboratory (CSHL) Professor Adrian Krainer showed this brutal disease can be treated by tweaking a process called RNA splicing. This breakthrough resulted in Spinraza, the first effective treatment...
Tag: <span>RNA drugs</span>
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Enabling safe delivery of RNA drugs to the lungs
HOKKAIDO UNIVERSITY IMAGE: A SUSPENSION OF LIPID NANOPARTICLES SYNTHESIZED FROM THE NOVEL LIPOMER AA03-DL-10 (PHOTO: TAKUYA ISONO). CREDIT: TAKUYA ISONO Testing a large library of compounds reveals an easy-to-make lipid that can carry genetic code into the lung to treat disease. Hokkaido University researchers in Japan created and tested a library of lipid-based compounds to...
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Can a new protein bring RNA drugs where they can’t reach?
Amber Tong Senior Editor Sue Dillon remembers the uncertainty when she first left J&J to start her own biotech around a new kind of molecules called centyrins. Sue Dillon J&J had already licensed the tech, which was developed by her co-founder Karyn O’Neil at the pharma giant’s Centyrex unit, to two companies for use in CAR-T and...