A team led by Dr. Shu Yilai of Fudan University in Shanghai used the virus as a vector to add alternative DNA to cells in the child’s inner ear, which can capture vibrations and transmit sound to the brain, thereby restoring hearing.
This is the first case in the world where natural hearing pathways have been successfully restored through gene therapy.
With this gene therapy, some deaf children have significantly improved their hearing, and some children are even able to hear sounds as low as 50 to 55 decibels, which is about the level of sound for normal conversation.
This study not only successfully restored hearing in participating children, but also marked an important breakthrough in the field of gene therapy in medicine.
Specific steps:
1. Select the appropriate viral vector: The researchers selected a specific virus to use as a vector to deliver new DNA to target cells.
2. Extract and prepare DNA: The researchers extracted a piece of alternative DNA that encodes a protein called otoferlin. This protein plays a key role in normal hearing.
3. Combination of DNA and virus: Combine this alternative DNA with the viral vector to form a gene therapy agent that can be injected into the human body.
4. Injection of gene therapy agent: This gene therapy agent is surgically injected into the child’s inner ear cells.
5. Cell uptake and expression: Once the gene therapy agent is injected, the target cell absorbs this new DNA and begins to produce otoferlin protein according to its instructions.
6. Restore hearing: The newly produced otoferlin protein allows inner ear cells to normally capture sound vibrations and transmit them to the brain, thereby restoring hearing.
Notes:
This treatment is only suitable for congenital deafness caused by a lack of the otoferlin protein.
Due to the use of viral vectors, there is a risk of an immune response that may affect the effectiveness of treatment.
Detailed report: https://technologyreview.com/2023/10/27/1082551/gene-treatment-deaf-children-hearing-china/
Original text:
https://twitter.com/i/status/1719582434189267222
