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Researchers from deCode Chemistry & Biostructures, Invitrogen, and Families of Spinal Muscular Atrophy have identified a protein that is a potential target for the treatment of SMA. DeCode had earlier developed a class of compounds, C-5 substituted quinazolines, that increase expression of the protein known as the survival motor neuron (SMN) protein (C&EN, May 7, 2007, page 19). But until the recent finding (ACS Chem. Biol., DOI: 10.1021/cb800120t), scientists didn't understand the mechanism behind the increase. "This discovery gets to the level of really understanding how SMN deficiency can be corrected in the cells of the body," says Jill Jarecki, research director at FSMA.
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