Gene Therapy for Inherited Deafness Shows Lasting Hearing Restoration Years After Treatment

An international clinical trial co-led by Mass General Brigham and China's Eye & ENT Hospital of Fudan University demonstrates that gene therapy successfully restored hearing in 90% of participants with a rare form of genetic deafness, with results published in Nature on Wednesday. The treatment, administered through injection, showed lasting improvements years after researchers first treated deaf children three years ago. Some patients improved dramatically, gaining the ability to hear whispers and normal conversation, with benefits extending even to adult participants including a 32-year-old. Study author Zheng-Yi Chen, a Mass Eye and Ear otolaryngologist, notes these represent the most promising gene therapy results for inherited deafness to date.

Clinical Trial Results and Efficacy of Gene Therapy

• ▪A clinical trial in China testing gene therapy for inherited deafness found 90% of participants reported significant improvement in their hearing after receiving an injection
• ▪The gene therapy for inherited deafness clinical trial results were published in Nature on Wednesday
• ▪The gene therapy for inherited deafness clinical trial represents the most promising results to date for this type of treatment
• ▪The gene therapy for inherited deafness improved hearing in some adult participants, including a 32-year-old
• ▪Researchers first injected deaf children with the gene therapy treatment for hearing restoration three years before the latest results were published

Mechanism and Patient Outcomes of the Hearing Restoration Treatment

• ▪The gene therapy for inherited deafness was administered through an injection
• ▪Some patients treated with the gene therapy for inherited deafness improved their hearing to the point where they can hear whispers
• ▪Zheng-Yi Chen is a study author of the gene therapy for inherited deafness research and a Mass Eye and Ear otolaryngologist

Perspective of Zheng-Yi Chen and Mass Eye and Ear researchers

• ▪The gene therapy for inherited deafness shows lasting improvements years after initial treatment administration
• ▪The gene therapy for inherited deafness clinical trial represents the most promising results to date for this type of treatment

Perspective of Patients with inherited deafness and their families

• ▪Gene therapy for inherited deafness enabled some previously deaf patients to hear whispers for the first time
• ▪Gene therapy for inherited deafness allowed some adult patients including a 32-year-old to regain hearing ability