Therapeutics

Our understanding of synaptic transporter structure, function and regulation affords us with the opportunity to translate this knowledge to the arena of drug discovery, where leads for novel therapeutics can be developed and tested in vitro and in vivo. For example, we have developed a novel assay that permits a high-throughput screening approach to identify modifiers of acetylcholine signaling, targeting the presynaptic choline transporter (CHT). We have used this assay to identify multiple classes of novel molecules that appear to regulate the function of CHT and may provide new opportunities to offset changes in cholinergic signaling that arise in dystonia, Parkinson’s disease, neuromuscular, and autonomic disorders. Acting in collaboration with biotechnology and drug development companies, we are also providing critical expertise and efforts in their drug discovery projects. As we proceed further in our understanding of the contribution made by novel transporter regulators, we expect to embark on new efforts, both in house and with industry collaborators, to develop leads for novel medications.