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Patisiran RNAi Therapeutic Mechanism
Description
This template compares the disease mechanism of transthyretin amyloidosis with the mode of action of the siRNA drug patisiran, tracing wild-type and mutant TTR genes through transcription, translocation, and translation. In the treatment panel, patisiran directs RISC to degrade both wild-type and mutant TTR mRNA, reducing malfunctioning TTR tetramer accumulation compared to the untreated mechanism.
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