Postdoctoral Associate University of Florida College of Pharmacy Gainesville, Florida
Severe hearing impairment affects over 5% of the worlds’ population due to malfunctional hair cells in inner ears. Half of them is resulted from pathologic mutations in key regulators in auditory signaling pathways like TMC-1, MYO7A in hair cells in inner ear. Genetic disruption of pathologic alleles has been demonstrated as an effective method for the early prevention of progressive hearing loss. However, we are still lacking a safe, effective genome editing toolkit and the associated delivery method to transport the promising gene therapeutics into inner ear for hearing rescuing. Emerging exosome-based hearing therapeutics have demonstrated the high biocompatibility, bioavailability of exosomes and the associated biomolecular exchange between a variety of functional cells in inner ear. In this presentation, we presented the discovery of new gRNAs targeting Myo7a pathologic alleles in Shaker-1 mouse model and demonstrated electro-transfection of CRISPR/Cas9 ribonucleoprotein into exosomes for exosome-based inner ear gene therapy in vivo.
Learning Objectives:
Upon completion, participant will be able to understand the allele-specific genome editing and its application in gene therapy.
Upon completion, participant will be able to learn the design of allele-specific gRNA designs and characterization methods.
Upon completion, participant will be able to acquire the mechanism of electro-transfection and packaging capacity for exosomes.
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