Abstract 1646: In vivo target validation in orthotopic hepatocellular carcinoma model using formulated siRNA
| Autor: | Ashwin Gollerkeri, Shilu Wu, Catherine Pachuk, Baisong Liao, De-Min Zhu, Chunrong Wang, Debra Kellner, Marion Molina |
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| Rok vydání: | 2011 |
| Předmět: | |
| Zdroj: | Cancer Research. 71:1646-1646 |
| ISSN: | 1538-7445 0008-5472 |
| DOI: | 10.1158/1538-7445.am2011-1646 |
| Popis: | Hepatocellular carcinoma (HCC) is the sixth most common cancer worldwide and its incidence continues to increase. Although there are approved therapies for HCC, the prognosis remains poor. The 5-year survival rate is < 5%. Nucleic acid-based therapeutics such as RNAi may improve patient outcome. Nevertheless, target identification/validation for HCC and delivery of siRNA are major challenges facing the development of HCC-specific RNAi based therapeutics. To enable target validation in vivo using RNAi, we have developed nanolipid siRNA formulations for the in vivo delivery of siRNA to orthotopic tumors. PK/PD studies in model systems demonstrate that up to 80% knockdown of target protein is achievable within tumors and that significant knockdown persists for at least 10 days following a single dose of formulated siRNA. Efficacy studies using similar formulations of siRNAs against potential HCC tumor targets, including mTOR, will be presented. Citation Format: {Authors}. {Abstract title} [abstract]. In: Proceedings of the 102nd Annual Meeting of the American Association for Cancer Research; 2011 Apr 2-6; Orlando, FL. Philadelphia (PA): AACR; Cancer Res 2011;71(8 Suppl):Abstract nr 1646. doi:10.1158/1538-7445.AM2011-1646 |
| Databáze: | OpenAIRE |
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