New Strategy to Identify Small Molecule Therapies: Usher Syndrome III Case Study

 

Characterized by progressive deafness, blindness and variable balance disorder, Usher syndrome type III (USH3) is caused by destabilizing mutations in the gene encoding the clarin-1 protein (CLRN1).

This webinar details a successful new strategy to identify small molecule therapies to mitigate the hearing loss associated with these mutations, highlighting the promise of this discovery approach in the quest for drugs to treat other protein-destabilizing monogenic disorders.

Funded by the Usher III Initiative Research Consortium, the work described here was carried out by collaborators at Case Western Reserve University, University Hospitals Cleveland Medical Center and Charles River.

Presenters

  • Christopher J. Lock, BSc, PhD, Senior Scientist, Charles River
  • Yoshikazu Imanishi, PhD, Assistant Professor, Case Western Reserve University

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