Gene therapy for genetic hearing loss has become a clinical reality in the past two years, with five active clinical trials worldwide aiming to treat otoferlin-related hearing loss. In this presentation, we will review the scientific background of gene therapy, compare gene therapy modalities, describe preclinical progress in treating various forms of genetic hearing loss, and finally discuss preliminary results from the current clinical trials. We will also review the scientific, clinical, and ethical challenges in implementing gene therapy for hearing loss, and discuss how to approach patient care in the era of gene therapy.
Learning Objectives:
- Understand how gene therapy works.
- Describe the benefits and potential risks of gene therapy.
- Understand the barriers to broadly implementing gene therapy for hearing loss.
- Learn how to counsel patients about the option of gene therapy.
