Special Session: Gene TherapyClinical Challenges and Opportunities for Implementing Gene Therapy for Hearing Loss The goal of identifying the molecular etiology of permanent hearing loss is to improve human health through better therapies. Although clinical guidelines recommend genetics evaluation for all children with confirmed permanent hearing loss, there is not universal access to genetics evaluation among these children due to numerous barriers. Sensorineural hearing loss and auditory neuropathy spectrum disorder have typically been considered disorders which may be rehabilitated but not “cured.” The advent of gene therapy for inner ear disorders will reframe the concept of permanent hearing loss. In order to offer gene therapy more broadly to patients, a significant investment in research will be required to understand the molecular mechanisms of genetic hearing loss and to further develop gene therapy technology. Changes in clinical practice will be needed to improve timely diagnosis of hearing loss and to facilitate access to genetics services in order to incorporate gene therapy as a therapeutic alternative. In addition, there is a role for continued advocacy on behalf of patients with hearing loss.
Marci Lesperance is a Professor of Otolaryngology-Head and Neck Surgery at the University of Michigan and served as Division Chief for 15 years. She completed otolaryngology residency at the University of Michigan in 1994. Dr. Lesperance then completed a one-year research fellowship in the Laboratory of Medical Genetics at the National Institute of Deafness and Other Communication Disorders (NIDCD), followed by a Pediatric Otolaryngology fellowship at Children’s National Medical Center in Washington, DC. Dr. Lesperance’s research has been funded by NIH and outside foundations, and it focuses on the molecular genetics of hearing loss and using genetics to improve patient care. She has served as Chair of the Board of Scientific Counselors for the NIDCD. She has served as Surgeon in Chief for the Children’s and Women’s Hospital, and as Program Director for the University of Michigan’s Pediatric Otolaryngology Fellowship. She has held leadership roles in the American Society of Pediatric Otolaryngology and was a member of the Otolaryngology Residency Review Committee for the Accreditation Council for Graduate Medical Education. Dr. Lesperance is the Pediatric Otolaryngology section Editor for Cummings Otolaryngology-Head and Neck Surgery, 6th through 8th editions. Currently, she serves as Associate Chief Clinical Officer for Children’s Specialty and Women’s Services for Ambulatory Care Services at Michigan Medicine.
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