A new gene therapy implemented at a US hospital has allowed an 11-year-old boy to hear for the first time.
A gene therapy of the protein Otoferlin, which is encoded by the OTOF gene, managed to reverse a certain type of congenital deafness and allow 11-year-old Aissam Dam to hear for the first time in his life. It is expected to be tested in younger children soon.
Aissam Dam is an 11-year-old boy (the boy in the photo) who was born deaf and had never heard anything. He lived in a poor community in Morocco, and communicated with a self-invented sign language. He had never been to school.
Last year, after moving to Spain, his family took him to a hearing specialist, who made a surprising suggestion: Aissam might be eligible for a clinical trial using a new gene therapy.
On October 4, Aissam was admitted to the Children's Hospital of Philadelphia in the US, becoming the first person in the United States to receive gene therapy for congenital deafness. The treatment was successful, and a child who knew nothing about sound discovered a new world.
The issue affects thousands of people living with hearing loss, which is defined as a disability. His is an extremely rare form, caused by a mutation in a single gene, Otoferlin. Otoferlin deafness it affects about 200.000 people worldwide.
The goal of gene therapy is to replace the mutated otoferlin gene in patients' ears with a functional gene.
