The article titled “Gene Therapy Allows an 11-Year-Old Boy to Hear for the First Time” was featured in The New York Times in January 2024, broadcasting an interesting story within the realm of popular press.

Kolata, G. (2024, January 23). Gene Therapy Allows an 11-Year-Old Boy to Hear for the First Time. The New York Times.
https://www.nytimes.com/2024/01/23/health/deaf-gene-therapy.html

Aissam Dam, an 11-year-old boy born deaf in Morocco, experienced an experimental gene therapy at the Children’s Hospital of Philadelphia, becoming the first in the United States to be treated for congenital deafness caused by a rare mutation in the otoferlin gene. A study supported by Lilly and Akouos, Aissam’s has since made excellent progress, such as having near-normal hearing in the treat ear two months after the therapy.

This safe and effective therapy involved delivering the hair cells of the cochlea with a harmless virus that carried new otoferlin genes. With Aissam successful outcome, more cases have been reported for further studies. Specifically, focusing on younger children, including children from Taiwan and the United States. Other global efforts in China and Europe are also underway, targeting younger children with bilateral hearing loss, supported by various organizations and companies.

In a 2021 study titled “Overloaded Adeno-Associated Virus as a Novel Gene Therapeutic Tool for Otoferlin-Related Deafness,” published in Frontiers in Molecular Neuroscience, researchers explore the potential of gene therapy for specific types of genetic hearing loss. The focus is on cases where the inner ear structure remains intact. The experiment involves using overloaded adeno-associated viruses (AAVs) to deliver full-length coding sequences of the otoferlin gene in mice models. The study’s findings suggest that employing a single overloaded AAV vector for gene therapy could be a promising approach for treating certain forms of deafness, offering advantages over other methods. This scientific review article supports the “popular press” article information, and contributes to more future research on gene therapy and its overall benefits.

Gene therapy involves modifying or replacing genes within an individual’s cells to treat/prevent certain diseases and conditions. It has the possibilities to treat genetic disorders in a persistent and curative manner by directly attacking the genetic foundation of diseases. Therefore, gene therapy represents the significance of genetics knowledge and technology used in medicine.

Kolata, G. Gene therapy allows an 11-year-old boy to hear for the first time. The New York Times, https://www.nytimes.com/2024/01/23/health/deaf-gene-therapy.html. (2024)

Rankovic, V. et al. Overloaded adeno-associated virus as a novel gene therapeutic tool for otoferlin-related deafness. Frontiers, 13, https://doi.org/10.3389/fnmol.2020.600051 (2021)