Drug pipeline
Full profile accessExplore the programs pursuing this target and their development progress.
- Drug candidates
- Developers
- Development stage
Target intelligence / Profile preview
Poly [ADP-ribose] polymerase 1 (PARP1) mRNA is the messenger RNA transcript that encodes the PARP1 enzyme, a critical factor in the cellular response to DNA damage [NCBI Gene ID: 142]. The PARP1 protein is primarily involved in identifying single-strand DNA breaks and initiating the base excision repair pathway, but it also plays roles in chromatin remodeling and cell death pathways like parthanatos [PubMed: 29128124]. In oncology, PARP1 mRNA is a target for gene-silencing therapies, such as small interfering RNAs (siRNAs) and antisense oligonucleotides (ASOs), which aim to deplete the protein to induce synthetic lethality in cancer cells with existing DNA repair defects, such as BRCA1/2 mutations [PubMed: 28677110]. This mRNA-targeting approach is distinct from traditional small-molecule PARP inhibitors, which bind to the protein's catalytic site; silencing the mRNA can potentially bypass resistance mechanisms or avoid the toxicities associated with "PARP trapping" on DNA [PubMed: 31533938]. Beyond cancer, the regulation of PARP1 mRNA is investigated in inflammatory and neurodegenerative diseases where excessive PARP1 activity leads to energy depletion and cell death [PubMed: 25635911]. Consequently, PARP1 mRNA represents a strategic target for precision medicine and nucleic acid-based therapeutics.
RNA interference (RNAi) or antisense-mediated degradation of the mRNA transcript to prevent translation of the PARP1 protein.
1 more in the full profile.
Beyond the preview
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Explore the programs pursuing this target and their development progress.
Follow the clinical studies evaluating therapies directed at this target.
Compare approaches across drug candidates, modalities, and indications.
Investigate the research and source evidence behind target biology and development.
Explore patent activity around therapies and technologies addressing this target.
Connect target biology, drug development, and emerging evidence in your research.
See how Gosset can support your research on Poly [ADP-ribose] polymerase 1 mRNA (PARP1 mRNA).