Chinese scientists use genetic therapy to regain the sound of deaf mice

Chinese scientists use genetic therapy to regain the sound of deaf mice

In China, 2-3 out of every 1,000 newborns have hereditary congenital deafness. This genetic disease has not been well treated, either relying on hearing aids and cochlear implants, or Gradually adapt to this "silent life." However, with the rise of gene therapy, these congenital deaf patients may one day be able to regain the ability to "capture the sound"!

In yesterday's "Natural Biotechnology" magazine, researchers from Harvard Medical School and Boston Children's Hospital published two articles using a synthetic viral vector carrying a therapeutic gene transplanted into the ear of a mouse. The hearing of the treated mice recovered to a level of 25 decibels [1, 2]! What is the concept of 25 decibels? Probably the volume of a person who whispers softly in our ears, which is a great improvement!

中国科学家借助基因治疗让失聪小鼠重获声音

Cochlear implant

Hereditary congenital deafness sounds like a disease, but genetically, scientists have discovered more than 100 related genes, each representing a different type of deafness. In this study, the researchers selected the deaf mice that were caused by mutations in the Ush1c gene, which accounted for more than half of all. Mutations in the Ush1c gene cause a protein called a harmonin to lose its function, causing damage to the ear hair cells that should receive sound and transmit acoustic signals to the brain, causing severe hearing damage.

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In this study, the researchers used a synthetic adeno-associated virus vector code-named Anc80L65. The adeno-associated virus vector is stable, safe, and highly targeted. It is a common type of viral vector used in gene therapy. . Why choose artificial synthesis? Is the natural adeno-associated virus vector not easy to use? This is to say that in 2015, they did the research.

中国科学家借助基因治疗让失聪小鼠重获声音

Professor Jeffrey Holt

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