New drug targets are important to study to find new and more effective therapies to alleviate human disease. Currently, RNA and associated RNA-binding proteins are significantly underutilized as a potential target for drug development: most likely because there exists a lack of basic knowledge about how one should design a molecule to target these structures. In our research, we are developing a small molecule to interfere with nucleocapid protein 7, NCp7, and its association with viral HIV RNA. In order for HIV to mature properly, NCp7 must bind to HIV RNA and protect it from degradation. Interfering with this process is a strategy to halt with HIV's progression and could become a valuable new treatment for HIV infected individuals. We have continued to develop our small molecule and are we are engaged in the necessary translational work to move our molecule toward a clinical trial. Over the past year, we have determined that the molecule is orally bioavailable and not toxic to rats. Further studies on the safety of this molecule will continue along with more investigations into the mechanism of action.

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Support Year
11
Fiscal Year
2015
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Indirect Cost
Name
U.S. National Inst Diabetes/Digst/Kidney
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Ugaonkar, Shweta R; Clark, Justin T; English, Lexie B et al. (2015) An Intravaginal Ring for the Simultaneous Delivery of an HIV-1 Maturation Inhibitor and Reverse-Transcriptase Inhibitor for Prophylaxis of HIV Transmission. J Pharm Sci 104:3426-39
Pokorski, Jonathan K; Appella, Daniel H (2012) Combinatorial Synthesis, Screening, and Binding Studies of Highly Functionalized Polyamino-amido Oligomers for Binding to Folded RNA. J Nucleic Acids 2012:971581
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Wang, Deyun; Iera, Jaclyn; Baker, Heather et al. (2009) Multivalent binding oligomers inhibit HIV Tat-TAR interaction critical for viral replication. Bioorg Med Chem Lett 19:6893-7
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