Novel Targeted Gene Therapy in Hearing Loss Diseases

The specialized structure of inner ear and the associated biological barriers to protect the bloodborne agents from the inner ear fluidics pose the great hurdles in the non-invasive delivery of therapeutics into hair cells in the cochlear to treat the malfunctional cells. The new discovery of hair cell-related receptors and key protein regulators in the regeneration of hair cells is boosting the development of novel gene therapy in hearing loss diseases. Meanwhile, exosomes derived from stem cells have been demonstrated to show a great hearing rescuing effect reported from different case studies. Utilizing exosomes to treat hearing impairment is envisaged as a good alternative to viral vectors which are mostly utilized in the gene therapy in hearing loss diseases. Besides, an automated platform for a fast, large scale production of functional gene therapy loaded exosomes is also highly demanded to cater to the future clinical need. The talk will focus on the most recent advance of gene therapy in hearing loss diseases, and the novel targeted gene therapy to overcome the hurdles in the associated unmet clinical needs.