About half of the cases of deafness are caused genetically. And treatment for these cases is very limited. Well, it was. In the past, a few technologies appeared that allow to treat the problem itself – genetic mutations in a deaf patient that cause him to be deaf.
The results of the study - Source: Gao et al./Nature 2017/
One of these mutations is the mutation of the Tmc1 gene. A problem with this gene causes the patient to gradually lose working hair cell inside the inner ear that function as receptors for the human hearing organ. If a human or for example a lab mouse has just one mutated copy of the Tmc1 gene it will lead to deafness over time. Unless something is done about it. That’s what David Liu and his colleagues decided to do and their study was published a few days ago by the Nature magazine.
Source: Broad Institute YouTube Channel
Liu’s team’s research is one of the first uses of a gene editor to treat deafness in animals. They did it with mice so it is still unsure whether it will work in humans as well. But the researchers believe that their research could become the base for development of new types of treatment for people with deafness caused by genetics. Liu and his team used CRISPR/Cas9. With this gene editor, they decided to get rid of the mutated gene leading to hearing loss in mice because of a mutation called Beethowen. The CRISPR system works as a targeting device and the Cas9 are essentially scissors that cut the DNA at the desired place. The problem here is that the mutated and correct version of the Tmc1 gene differs in just one DNA base. To ávoid getting rid of the correct version of the gene the scientist used a special lipid container.
The scientists injecting this version of the CRISPR/Cas9 editor into the inner ear of young mice with the Beethowen mutation and waited to see what will happen. After eight weeks these hair cells had grown just like they did for healthy mice. And a detailed study confirmed that the hearing for the treated mice improved significantly.
Sources:
I knew i have heard "crispr" somewhere before, them i remembered...The Blacklist season 4, that aside..so this tech really exist and if so its a giant step for humanity and genetically induced conditions
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CRISPR is seriously amazing, I've written about it in a few articles before as well. It's pretty much THE tool we have been waiting for to get genetic modification really rolling.
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i guess the future is here, this creates hope for diabetic patients, even sickle celled patients might see change coming
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