In a remarkable medical breakthrough, deaf children in China are experiencing the wonder of hearing for the first time thanks to gene therapy. This innovative treatment is proving effective in restoring the natural hearing system of deaf children, opening doors to a world of previously inaccessible sounds. In this article, we will explore how gene therapy is transforming lives and offering hope to thousands of children around the world.

The Miracle of Gene Therapy

Imagine the moving story of Yiyi, a deaf child who, after years of silence, can finally hear her mother and even dance to the music. What makes this story even more incredible is that Yiyi was born completely deaf. However, thanks to gene therapy, that reality has changed for her and her family.

The gene therapy, developed and refined by a team of Chinese scientists led by Dr. Yilai Shu of Fudan University in Shanghai, has the potential to be a game changer in restoring natural hearing in deaf children. During the procedure, doctors use a virus to add replacement DNA to cells in Yiyi's inner ear, which capture vibrations and transmit them to the brain as sound.

In less than a month, Yiyi began hearing with the treated ear for the first time. Now, she can enjoy activities that were previously impossible, like hearing the bell that signals the end of nap time at school. Before, she depended on others to receive this information.

A Historic Feat

The success of gene therapy in China is truly remarkable, given that, until now, no medication has been able to improve hearing. This achievement is a milestone in medical research and could open doors to the development of innovative treatments for other deaf conditions.

Dr. Yilai Shu and his team began treatments in December last year, after years of research and testing on mice and guinea pigs. The challenge was to find an effective way to deliver genetic material to the inner ear. However, the results speak for themselves, with several children, including Yiyi, largely recovering their hearing.

The Benefits of Gene Therapy

Gene therapy is not a panacea for all types of deafness. Therefore, it applies specifically to cases of congenital deafness caused by a defect in a gene that produces a protein called otoferlin. This protein plays a crucial role in transmitting information from the inner ear to the brain. Therefore, the absence of otoferlin impairs the ability to hear.

It is important to note that defects in the otoferlin gene are responsible for only 1% to 3% of congenital deafness cases, making it a rare condition. However, success in restoring hearing in children with this rare condition is stimulating research into genetic treatments for other causes of deafness.

How the Treatment Works

Genetic treatment involves adding a functional copy of the otoferlin gene. In other words, due to the considerable size of this gene, which contains around 6,000 DNA letters, it is divided into two parts. Each part is packaged separately in millions of copies of a harmless virus. Dr. Shu then carefully injects these viruses into the children's ears, where the two sections of DNA recombine to create a complete gene capable of producing the missing otoferlin protein.

Although gene recombination may be inefficient in other contexts, gene therapy results demonstrate its surprising effectiveness. Therefore, after treatment, the children went from having a complete lack of hearing to being able to hear sounds at regular conversational levels.

The Effects on Children's Lives

The results of gene therapy are truly inspiring. Many of the children who participated in the studies are experiencing significant changes in their lives. Therefore, some of them, who had never spoken before, began to pronounce simple words like “mommy” and “daddy” after the treatment. This highlights the importance of treating children at a young age when speech development is in full swing.

The Potential of Gene Therapy in Hearing

Gene therapy represents a remarkable advance in the treatment of deafness. Although cochlear implants have been a huge success, they have their limitations. With these implants, patients can hear sounds, but the subtlety of the sounds is often lost. Gene therapy has the potential to offer hearing closer to normal, that is, allowing children to experience the richness of sounds that often goes unnoticed.

The Future of Gene Therapy

The success of gene therapy in China is generating excitement among researchers and biotechnology companies around the world. Therefore, several Western companies have already begun clinical trials for gene therapy treatments targeting the otoferlin gene, looking for ways to expand the reach of this innovative therapy.

Conclusion

Finally, gene therapy is transforming the lives of deaf children in China and offering hope to many others around the world. Effective treatment of genetic defects that cause congenital deafness is a major advance in medicine. As such, its successful implementation is a testament to the power of medical research and human determination.

With information @ MIT Technolgy Review