Developing Site Specific DNA Binding Agents Using Novel Purine Analogs

Indianapolis, IN

We are using customized, synthetic purines as chemical building blocks to develop site-specific DNA binding agents with therapeutic potential. The specific prototype we aim to develop has been designed to span six base pair sequences in the minor groove of DNA, binding selectively to C-G base pairs. We envision this agent to be potentially useful in the treatment of Fragile X Syndrome, a trinucleotide repeat disorder resulting in a large number of CGG repeats within certain genes. The synthesis of this prototype drug molecule uses a novel purine amino acid monomer platform. The synthetic route we have is, mostly, a. composed of well known, high-yielding reactions, and b. modular and thus capable of producing families of potential drug candidates.