Research
We leverage chemical approaches to develop small molecules that target nucleic acids, with the goal of advancing both basic science discoveries and novel therapeutics for diseases with no cure.
The Schneekloth laboratory focuses on the identification, characterization and development of small molecules that target nucleic acids.
Our efforts leverage chemical approaches to
- design probe molecules that inform basic science discoveries, and
- help advance nucleic acid-targeting molecules toward mechanistically novel therapeutics for diseases with no cure, including cancers, neurodegenerative diseases, and infectious diseases.
This work encompasses development of new assay technologies (including high-throughput screening and biophysical assays) for ligand discovery/characterization, synthetic/medicinal chemistry, structure-guided design of ligands for nucleic acids, mapping ligand binding sites in human transcriptomes using photo crosslinking probes, biochemistry of nucleic acids and cell biological approaches.
A key technology is the use of a small molecule microarray (SMM) high-throughput screening platform to rapidly discover and profile the binding of molecules to structurally and functionally diverse nucleic acids relevant to cancer and infectious disease.
A particular strength of the laboratory has been to use synthetic chemical approaches in concert with internally developed novel screening technologies and high-throughput chemical biology to identify inhibitors of highly challenging, biologically valuable nucleic acid targets such as c-Myc, MYCN, and KRAS.