Gene therapy aims to fix a faulty gene or replace it with a healthy gene to try to cure disease or make the body better able to fight disease.
It holds promise as a treatment for a wide range of diseases, such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS
Recent Development
Gene therapy and genome editing are potential treatments for hereditary hearing loss.
Adeno-associated virus (AAV) is a common vector used in gene therapy due to its safety and ability to infect various cells.
A recent study published in Nature Medicine by Chinese researchers from Fudan University demonstrated promising results using gene therapy for genetic deafness involving the OTOF gene.
The OTOF gene encodes otoferlin, a transmembrane protein belonging to the ferlin protein family, protein associated with genetic human deafness
The study showed significant improvement in hearing in patients treated with AAV vectors carrying the healthy OTOF gene, with no severe side effects observed.
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