Virus containing certain genes is injected into the ear
The CHORD experiment started in May last year. Aim, OTOF mutation The aim was to examine whether the gene therapy called DB-OTO works in cases with Opal, received an infusion containing the harmless AAV1 virus.
This operation is performed under general anesthesia. cochlea (ear cochlea) was performed by injection. Thus, the genetic material of the virus was transferred to the cells. While gene therapy was applied to the child’s left ear, an implant was also placed.
The essay consists of three parts featuring three children, including Opal. The sick children received a small dose of the gene therapy for just one ear. It is stated that this reduces the side effects. The process is only 20 minutes lasted.
The results are very successful
Approximately 4 weeks later Opal, even when the cochlear implant in the left ear is turned off He started to react to sounds. Doctors subsequently monitored the situation and found significant improvements. It was determined that after the 24th week the child could hear whispers.
Professor Manohar Bance, an ear surgeon at the University of Cambridge and head of the study, said: your results are amazing He said it was much better than he expected and continued: “Gene therapy has been the future of otology and audiology for many years, and I am very excited that it is finally here. It is a treatment for the inner ear and many types of hearing loss.” The beginning of a new era for gene therapies We hope it will.”
Opal was born completely deaf due to auditory neuropathy, a rare genetic disorder. Although the ear perceives sound normally in auditory neuropathy spectrum disorder, It has problems sending these signals to the brain. This problem can cause problems with distinguishing sounds and clarity, as well as deafness. Every disease One in 10 thousand babies It is stated that it was seen. As we mentioned before, the disease is caused by a mutation in a single OTOF gene. The OTOF gene produces a protein called otoferlin. This protein plays a role in communication with the inner hairs of the ear and the auditory nerve.
Research continues
In the first phase of the research, a small dose is given to one ear of the patients. Then, in the second stage, higher doses of gene therapy are used in only one ear. It is important here to prove that the starting dose is safe. In the third stage, after determining its safety and effectiveness, gene therapy is continued in both ears with the selected dose. It is stated that follow-up examinations of treated patients will continue for five years.
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