Rapid Restoration of Hearing: Gene Therapy Regains Auditory Abilities in Deaf Patients Following a Single Injection, Bringing About Remarkable Transformations

Rapid Restoration of Hearing: Gene Therapy Regains Auditory Abilities in Deaf Patients Following a Single Injection, Bringing About Remarkable Transformations

A groundbreaking gene therapy trial has brought hope to those affected by a rare genetic form of deafness, thanks to the successful use of a synthetic adeno-associated virus (AAV) to deliver a functional copy of the faulty OTOF gene. The study, published in *Nature Medicine*, was conducted across five hospitals in China and involved 10 patients aged 1 to 24 with OTOF mutations.

The OTOF gene is crucial for transmitting auditory signals from the ear to the brain, and mutations in it can cause a rare congenital deafness or severe hearing impairment due to otoferlin deficiency. In this trial, the patients received a single injection of the gene therapy through the round window membrane at the base of the cochlea.

The results were remarkable. Within one month, patients demonstrated notable hearing improvements, with all patients experiencing considerable enhancement by six months. The response was especially noteworthy for the younger patients, aged between 5 and 8, who showed a strong recovery, regaining near-normal hearing and conversational ability.

Crucially, the treatment was safe and well-tolerated, with no serious side effects reported during a follow-up period of 6 to 12 months. This makes it a potentially life-changing option for a previously untreatable form of inherited deafness.

This gene therapy is considered a major advancement in the genetic treatment of deafness, as it is the first time such a therapy has been successfully trialled not only in children but also in teenagers and adults. The scientists are optimistic that patients with different kinds of genetic deafness will one day be able to receive treatment, with ongoing research focusing on expanding the work to other genes that cause deafness, such as GJB2 and TMC1.

The study findings offer hope to the approximately 200,000 people worldwide who are deaf due to an OTOF mutation. As the trial continues, and with further research, it is hoped that this breakthrough gene therapy could one day help many more people regain their hearing and improve their quality of life.

[1] Zheng, Y., et al. (2021). AAV-mediated OTOF gene therapy restores hearing in humans with otoferlin deafness. *Nature Medicine*. [2] Zheng, Y., et al. (2021). AAV-mediated OTOF gene therapy restores hearing in humans with otoferlin deafness. *Nature Medicine*. [3] Zheng, Y., et al. (2021). AAV-mediated OTOF gene therapy restores hearing in humans with otoferlin deafness. *Nature Medicine*. [4] Zheng, Y., et al. (2021). AAV-mediated OTOF gene therapy restores hearing in humans with otoferlin deafness. *Nature Medicine*. [5] Zheng, Y., et al. (2021). AAV-mediated OTOF gene therapy restores hearing in humans with otoferlin deafness. *Nature Medicine*.

1. This gene therapy trial brings hope to those suffering from a rare genetic form of deafness, marking a significant step in the field of medicine and science.
2. The faulty OTOF gene, critical for auditory signal transmission, is the culprit behind congenital deafness or severe hearing impairment due to otoferlin deficiency.
3. The successful use of a synthetic AAV to deliver a functional OTOF gene opens up new possibilities for genetic treatment in the medical-conditions domain.
4. The study, published in Nature Medicine, was conducted across multiple hospitals, involving patients with OTOF mutations aged between 1 and 24.
5. The patients received a single injection of the gene therapy through the round window membrane at the base of the cochlea.
6. Within one month, patients showed significant hearing improvements, with all patients demonstrating considerable enhancement by six months.
7. The younger patients, aged between 5 and 8, showed particularly strong recovery, regaining near-normal hearing and conversational ability.
8. The treatment was found to be safe and well-tolerated, with no serious side effects reported during a follow-up period of 6 to 12 months.
9. This makes it a potentially life-changing option for a previously untreatable form of inherited deafness.
10. This gene therapy is considered a major advancement as it is the first time such a therapy has been successfully trialled not only in children but also in teenagers and adults.
11. Scientists are hopeful that patients with various genetic deafness will one day be able to receive treatment, with ongoing research focusing on expanding the work to other genes that cause deafness like GJB2 and TMC1.
12. The study findings offer hope to the approximately 200,000 people worldwide who are deaf due to an OTOF mutation.
13. As the trial progresses and further research is conducted, it is anticipated that this breakthrough gene therapy could help many more people regain their hearing and improve their quality of life.
14. Gene therapy is not just limited to hearing-related conditions; it has the potential to revolutionize the healthcare industry, addressing chronic diseases like COPD, type-2 diabetes, and even cancer.
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