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PARP1 siRNA is a research-stage experimental modality consisting of small interfering RNA (siRNA) designed to silence the expression of the PARP1 gene, which encodes the Poly(ADP-ribose) polymerase 1 enzyme. PARP1 is a critical component of the DNA damage response, specifically involved in the repair of single-strand breaks via the base excision repair pathway. By utilizing RNA interference (RNAi) to degrade PARP1 mRNA, this modality effectively knocks down protein expression, thereby impairing DNA repair and sensitizing cancer cells to DNA-damaging agents or inducing synthetic lethality in cells with existing repair deficiencies, such as BRCA1/2 mutations. While not a proprietary drug asset, PARP1 siRNA is widely used in preclinical research to validate the therapeutic potential of PARP inhibition in various cancers, including non-small cell lung cancer, ovarian cancer, and prostate cancer, as well as in non-oncological conditions like diabetic peripheral neuropathy.
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