Eli Lilly-backed biotech grabs $100M to dispatch antibody-oligonucleotide conjugates after muscular dystrophy
Hold up your hand. Make a fist. Now open it. And again.
If you can do it fully and with ease, then the proteins in your hand are likely working properly. If you can’t then they may not be. In people with myotonic muscular dystrophy, something more atomic is going on.
In those folks, the problem is RNA. Certain base pairs repeat far beyond normal, up to 11,000 superfluous letters in some cases. The extended strands form “clumps.” Proteins misform and can’t function properly. They often allow one movement but not the reverse, a condition called myotonia that gives the dystrophy its name.
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