Chemical chaperones for refolding mutant SOD1

National Center For Advancing Translational Sciences

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Linked publications & trials

Abstract

The collaborative team aims to identify small molecule pharmacological chaperones (pharmacoperones) capable of improving folding of mutant superoxide dismutase 1 (SOD1). During this period, the collaborative team completed optimization of the high-throughput amenable assay and conducted a quantitative high-throughput screen of approximately 20,000 compounds. Additional assays to characterize the folding status of SOD1 have been developed and are staged for characterizing compounds identified via screening efforts. In addition, team members have synthesized several compounds that are critical for advancing the project.