Tag: RNA splicing
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How SKY-0515 Uses RNA Splicing to Lower HTT and PMS1
SKY-0515 modifies RNA splicing to lower full-length huntingtin and PMS1, potentially addressing both toxic-protein production and somatic CAG-repeat expansion.
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How Toxic RNA Drives DM1—and Why Del-desiran Was Designed to Silence It
DM1 is driven by toxic CUG-repeat DMPK RNA that sequesters MBNL proteins and disrupts alternative splicing. This explainer examines how del-desiran was designed to correct that mechanism—and why molecular improvement did not translate into a positive Phase 3 primary endpoint.