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 gene therapy.
On Oct. 4, Aissam was treated at the Children’s Hospital of Philadelphia, becoming the first person to get gene therapy in the United States for congenital deafness.
His is an extremely rare form, caused by a mutation in a single gene, otoferlin.
Otoferlin deafness affects about 200,000 people worldwide.
The goal of the gene therapy is to replace the mutated otoferlin gene in patients’ ears with a functional gene.
Persons:
Aissam, ” Aissam, “
Organizations:
Children’s Hospital of Philadelphia
Locations:
Aissam, Morocco, Spain, United States