Treatment of congenital deafness, CRISPR or into next-generation gene therapy?
December 22, 2017 Source: DeepTech Deep Technology
Window._bd_share_config={ "common":{ "bdSnsKey":{ },"bdText":"","bdMini":"2","bdMiniList":false,"bdPic":"","bdStyle":" 0","bdSize":"16"},"share":{ }};with(document)0[(getElementsByTagName('head')[0]||body).appendChild(createElement('script')) .src='http://bdimg.share.baidu.com/static/api/js/share.js?v=89860593.js?cdnversion='+~(-new Date()/36e5)];On Wednesday, an animal study published in the journal Nature revealed that the CRISPR system can be used to treat congenital hearing loss, a deafness caused by genetic defects.
Microvilli cells located in the cochlea are tissues responsible for transforming external sonic vibrations into signals that can be processed by the brain. Damage to microvilli cells can result in hearing loss. For example, this kind of thing happens when people are immersed in loud music for a long time.
Similarly, some specific gene defects can lead to similar results, and the Tmc1 gene is an example. Mutations in this gene express a mutated toxic protein that accumulates to kill cochlear villus cells. In humans and mice, as long as a single Tmc1 gene is inherited, it leads to progressive hearing loss and eventually to paralysis.
In this study, scientists packed the CRISPR-Cas9 system into microlipid vesicles and injected them into the inner ear of mice. This system specifically modifies and destroys the mutant Tmc1 gene in the cells of the ear. In the mouse samples that received the CRISPR "drug" injection, more mouse ear villus cells survived and re-growth after a few weeks. To test their hearing recovery, the researchers mounted the sensing electrodes on the head of the mouse and monitored their brain's response to external sounds. Compared to the treated mice, the control mice (not receiving real treatment) required a louder volume of sound to activate the brain's response. This study thus demonstrated the effect of this treatment on hearing repair in mice.
In fact, there are still a large number of genes in humans associated with congenital deafness. As we mentioned in the previous article, through this drug delivery system, we can use the highly extended CRISPR technology to treat congenital deafness in humans.
It is worth mentioning that the results were completed by a team led by Chinese biologist David Ruchien Liu from the Bode Institute in the United States. Liu Ruqian is studying how to use the CRISPR genome editing technology to optimize cell-based therapies, and was therefore selected as one of the top ten scientific figures of Nature in 2017.
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