Study provides insights into the action and specificity of splice-modifying drugs

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Other: Ian Birkby

Study provides insights into the action and specificity of splice-modifying drugs

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.