Special Session: Gene TherapyPediatric Otolaryngology in the Era of Gene Therapy for Deafness After decades of research focused on how to regenerate or replace hair cells, we now are at the advent of providing novel treatment to reverse what was thought to be a permanent hearing loss with the use of gene therapy. While the current application of gene therapy is narrow, limited to oterferlin-related auditory neuropathy, early results are promising, and the success of the early adopters inspires an era of new research, regarding how children learn to hear when provided acoustic hearing postnatally. How to identify potential candidates for otoferlin gene therapy and how to integrate this therapy into our toolbox will be an important area of development for our profession. Genetic testing is the key that opens opportunities for these important novel therapies. Expanding both access to genetic testing and our understanding of pathogenic variants in understudied populations will be important goals to providing hearing broadly for those children who can benefit from these groundbreaking therapies. Whether the early success of otoferlin gene therapy will be replicated in other forms of genetic hearing loss remains an open question.
Keiko Hirose is a tenured professor at Washington University and a pediatric otolaryngologist and surgeon-scientist. Her clinical practice is focused on the care of deaf children, cochlear implantation, and chronic ear disease. Her research program has established the importance of innate immunity in the inner ear, demonstrating the role of myeloid cells in congenital CMV infection, pneumococcal meningitis/sepsis, and surgical implantation of neural prostheses. She served as division chief of pediatric otolaryngology at WashU for fifteen years and has been an integral part of the auditory neuroscience community at the Central Institute for the Deaf. Nationally, she has taken part in the leadership of the Association for Research in Otolaryngology, serving on ARO Council and as ARO president in 2020. Dr. Hirose has served on the Research Advisory Board of the American Otologic Society (AOS), the Program Committee for the AOS and the American Society for Pediatric Otolaryngology (ASPO).
|
