Context:
Recently, a UK girl born deaf could hear unaided, after a gene-therapy treatment.
About the Patient and disease:
- She (Opal Sandy) had the therapy in her right ear, under general anaesthetic, and a cochlear implant put into her left ear.
- A cochlear implant is an electronic device that improves hearing.
- She is the youngest person to have their hearing restored after undergoing new gene therapy.
- She had a genetic form of auditory neuropathy caused by the disruption of nerve impulses traveling from the inner ear to the brain.
About the therapy:
- The gene therapy known as ‘DB-OTO’ is specifically designed for children with OTOF mutations.
- In this method, a safe virus is used to inject the patient with the functional gene.
- Auditory neuropathy can be caused by a fault in the OTOF gene.
- The OTOF gene produces a protein called otoferlin, which is crucial for the inner hair cells in the ear to communicate with hearing nerves.
- By inserting a functional copy of the gene into the ear, Regeneron’s “new era” gene therapy fixes the defect.
- The therapy uses a modified, harmless virus to deliver a working copy of the OTOF gene into these cells.
- Hearing loss caused by a variation in the OTOF gene is not commonly detected until children are two or three years old.
Significance of this therapy
- This successful case exemplifies the transformative potential of gene therapy in addressing genetic forms of hearing loss thus offering hope for affected individuals worldwide.
- This case also pave the way for further advancements in the related fields of medical genetics and gene therapy.
- Doctors in other countries are also exploring similar treatments for the OTOF gene mutation.
- Gene therapies are also being used to treat a few other diseases like: – an eye disorder called Leber congenital amaurosis and a muscle disorder called spinal muscular atrophy.
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