Bristol-Myers, Scripps research team touts a new way to control 3D architecture of genetic drugs
Whenever a patient is given one dose of an antisense drug — like the spinal muscular atrophy treatment Spinraza — chances are they are actually receiving thousands of distinct versions of the compound that differ in their spatial configuration. And that can be a real problem.
Given that the 3D architecture of biological molecules often impacts their function, scientists have suggested that “stereodefined systems,” where atoms’ arrangement in space are strictly controlled, might make drugs safer and more effective.
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