The new version of CRISPR technology is expected to cure Huntington's disease
February 28, 2018 Source: WuXi PharmaTech
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)];A new version of the genetic editing CRISPR/Cas9 technology for the treatment of Huntington Disease is safer and more specific than the earlier studies, and the findings are published in the recent issue of Frontiers in Neuroscience.
The idea of ​​using genetic editing techniques to cure diseases is getting closer to reality. For hereditary diseases, such as Huntington's disease (which is an incurable neurodegenerative disease caused by defective genes), resecting the DNA sequence that causes the disease seems to be the ultimate cure. However, researchers should ensure that this is the safest and most effective method before anyone cuts a patient's DNA.
â–²Researcher, Dr. Marta Olejniczak, Associate Professor, Institute of Bioorganic Chemistry, Poland (Source: Institute of Bioorganic Chemistry)
“In the study, we further improved the CRISPR/Cas9 technology by using the enzyme activity of Cas9,†said Dr. Marta Olejniczak, associate professor of the Institute of Bioorganic Chemistry, “Because Cas9 is recognized as safe. And with special effects, our method provides an effective treatment tool for Huntington's disease."
Huntington's disease is caused by an abnormal repetition of specific DNA sequences at the end of the Huntington gene. This defective mutant gene results in the production of toxic proteins that gradually accumulate and damage the patient's neurons. The disease usually begins in the 30s and 40s, and in the decades after the onset of the disease, the patient gradually loses the ability to move, talk or even think.
Huntington's disease is currently incurable. But researchers have tried many ways to suppress defective genes. This includes disrupting the production of toxic proteins by DNA and RNA based methods. Recently, researchers have also begun to use one of the most popular gene editing tools, CRISPR/Cas9, which is easier, faster, and more accurate than past tools.
To ensure that this technology is as safe and effective as possible, Dr. Olejniczak's team has tested a new Cas9 protein variant in a patient model of Huntington's disease. This version of Cas9 can cut a DNA strand instead of two DNA, which increases the accuracy with which Cas9 can edit specific DNA sequences.
Dr. Olejniczak said: "We demonstrated that Cas9 can excise repetitive DNA sequences, resulting in the inactivation of genes that cause Huntington's disease and halt the synthesis of toxic proteins. Our strategy is safe and efficient, with no sequence-specific side effects observed."
Of course, there is still much work to be done in the future for this destructive genetic disease, and further advances are needed, but this research is another important step in the possible treatments.
Reference materials:
[1] Scientists move closer to treatment for Huntington's disease
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