Rare disease day | the new CRISPR technology is expected to cure Huntington’s disease

Source: rare disease day | the new CRISPR technology is expected to cure Huntington’s disease

Gene editing CRISPR/Cas9 technology to the new version for the treatment of Huntington’s disease（Huntington Disease, than the early studies of version more security and more specifically, this study results published in the near future of the Frontiers in Neuroscience magazine.

Using gene editing technology to cure the disease idea is more and more close to reality. For genetic diseases, such as Huntington’s disease, which is a by the defective gene causes the incurable neurodegenerative disease, removal of disease-causing DNA sequence seems to be the ultimate cure. However, thein any people cut the patient’s DNA before, researchers need to ensure that this is the safest and most effective methodit.

▲head of research group, Poland bio-Organic Chemistry Institute of Professor Marta Olejniczak Dr. photo source: Institute of Bioorganic Chemistry）

“In the study, we use a Cas9 enzyme activity is a further improvement of the CRISPR/Cas9 technology,”head of research group, Poland bio-Organic Chemistry Institute of Bioorganic Chemistry）Associate Professor Marta Olejniczak Dr. said,“because Cas9 has been recognized to be safe and effects, our approach for Huntington’s disease to provide an effective therapeutic tool.”

Huntington by the Huntington gene to the end of the specific DNA sequence abnormalities caused by repeated. This defective mutations in the gene lead to toxicity of the protein produced, the protein gradually accumulated and damage to the patient’s neurons. This disease usually begins in three to four years old at the onset of the disease after several decades, patients gradually lose activity, talking and even thinking ability.

The current Huntington’s disease there is no rule of law more. But researchers have tried many methods to suppress the defective gene. This includes DNA-based and RNA methods to interrupt the toxic protein. Recently, researchers also started using the recent fiery genome editing tool-CRISPR/Cas9 technology, the technology than in the past the tools for more convenient, more fast, more accuracy.

In order to ensure that this technology is as safe and effective, Olejniczak Dr. the research team has been in a Huntington’s disease patient cell models to test a new kind of Cas9 protein variants.< strong>this version of Cas9 can cut a DNA Strand, rather than two pieces of DNA, which increases the Cas9 can edit a specific DNA sequence accuracyit.

Olejniczak said Dr.“we verified with Cas9 can cut the repetitive DNA sequence, thereby causing the caused by the Huntington’s disease gene inactivation and termination of the toxicity of protein synthesis. Our strategy is safe and efficient, we did not observe sequence-specific side effects.”

Of course, for this devastating genetic diseases the future there is still much work to be done, also need to further improve technology, but this study in the possible treatment method on another step important step.

References:

[1] Scientists move closer to treatment for Huntington’s disease

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Published at Tue, 28 Feb 2018 19:02:27 +0000