- Experimental gene therapy shows promise in treating hereditary hearing loss in children, allowing several children to hear after being born deaf.
- Preliminary studies in China and the United States have shown significant restoration of hearing in treated children, with one boy becoming the first in the US to receive gene therapy for congenital deafness.
- Gene therapy aims to replace a defective gene responsible for inner ear protein production, offering hope for treating hereditary deafness and potentially other types of deafness caused by genetic mutations.
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Experimental gene therapy has shown promise in treating hereditary hearing loss in children. In a study conducted in both China and the United States, gene therapy has allowed several children born with inherited deafness to hear. In one case, an 11-year-old boy named Aissam Dam of Spain, who was born unable to hear, underwent gene therapy at the Children’s Hospital of Philadelphia in October and became the first person in the US to receive the treatment for congenital deafness. Although the sound is muffled, Dam is now able to hear his father’s voice and sounds from cars on the road.
In a small study published in The Lancet, researchers documented significantly restored hearing in five out of six children treated in China. Another study conducted by Chinese researchers also showed similar results in two other children. The preliminary results from other research have also been positive, with a study sponsored by Regeneron Pharmaceuticals and Decibel Therapeutics showing improvements in a child under 2 years old.
Hereditary deafness affects 34 million children worldwide, with genes responsible for up to 60% of cases. Gene therapy is a promising approach for treating hereditary deafness, joining treatments already approved for other illnesses such as sickle cell disease and severe hemophilia.
To administer the gene therapy, surgeons at the Children’s Hospital of Philadelphia developed a minimally invasive endoscope that allowed for the surgical placement of the therapy directly into the inner ear. The therapy aims to replace a defective gene responsible for inner ear protein production, restoring hearing function.
While the experimental therapies currently target only one specific condition, researchers hope that this research could eventually help children with other types of deafness caused by genetic mutations. Despite the improvements seen in the treated children, questions remain about the long-term effectiveness and potential side effects of the therapy.
Some people have raised ethical concerns about gene therapy for deafness, arguing that it may be seen as a threat to Deaf communities and their use of sign language. However, researchers are moving forward with their work, emphasizing the potential benefits and the progress made in the field of gene therapy.