Gene therapy for deafness are we there now
embopress.orgMar 25
Eighteen-month-old Opal Sandy from Oxfordshire, England — who was born deaf due to defective OTOF genes — can now hear "almost normal for her age," including "soft sounds" such as whispers, thanks to a new gene therapy called DB-OTO.
Due to a faulty gene, Opal was born with auditory neuropathy, which disrupts nerve impulses that travel from inside the ear to the brain. As part of the DB-OTO trial, which lasted 16 minutes, she was infused with a working copy of the gene.
Recent gene therapy breakthroughs are not only exciting because they're helping children hear, but because these studies have resulted in no adverse effects. As scientists continue to cure deafness for these rare genetic disorders, these results will hopefully help further research for hearing-impaired people around the world.
These treatments, unfortunately, seem to be fully effective just for those born recently. Whether for deafness, muscular atrophy, or any disorder, current gene therapies for older people can only slightly mitigate the symptoms that have set in over long periods. More research should be conducted into fixing these disorders at all stages.