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Home / Science / The modified CRISPR gene editing tool could improve the therapies for HIV and sickle cell disease

The modified CRISPR gene editing tool could improve the therapies for HIV and sickle cell disease



  crispr
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City of Hope researchers may have found a way to sharpen the fastest, cheapest and most accurate gene editing technology, CRISPR-Cas9, to more successfully cut out unwanted genetic information.

This improved cutting ability could someday accelerate potential therapies for HIV, sickle cell disease and possibly other immune diseases.

"Our CRISPR-Cas9 design may be the difference between trying to cut a ribeye steak" A butter knife instead of cutting it with a steak knife, "said Tristan Scott, lead author of the study and research associate at the center for Gene Therapy of the City of Hope. "Other scientists have attempted to improve CRISPR cutting by chemical modification, but this is an expensive process and is equivalent to diamond coating a blade. Instead, we've developed a better pair of scissors that you can buy in any supermarket. "

The study, published on November 6, in Scientific Reports is the first time that scientists systematically review the lead Scott went through the mRNA sequence to change them and improve the CRISPR-Cas9 technology, Scott said Kevin Morris Lab of City of Hope has filed a patent application claiming this enhanced CRISPR-Cas9 design but the exact amount depends on the target site, said Scott.

This study is justified City of Hope, a globally recognized independent research and treatment center for cancer, diabetes, and other life-threatening diseases, is contributing The researchers are building essential building blocks for gene therapies with better-functioning plants to furnish witnesses.

The effects below could lead to "cleaner" results in experiments with cell and mouse models aimed at the development of new therapies because the target was "knocked out" more successfully. More pronounced results could accelerate new therapies from the laboratory to the bedside. Theoretically, the therapeutic product should have more successful sections, which could be reflected in improved therapy. Further research is required. The exact mechanisms why this change in the CRISPR system improves the editing of genes still has to be determined.

The researchers experimented on cells by making alterations to the "transactivating CRISPR RNA" (also known as "tracrRNA") derived from Streptococcus pyogenes bacteria and is part of the components that are used by genetic scissors (Cas9) correct gene sequence. Streptococcus pyogenes Cas9 is the most commonly used genetic scissors. Scientists used an RNA protein system because it produces a burst of activity that disappears about 1

2 hours after being introduced into the cell, meaning that the likelihood that the human genome will be inadvertently later edited after the fix is ​​done is less likely was, Scott said.

They found that the modified tracrRNA improved the silencing of certain genes by augmenting desirable mutations in the genetic material. In this study, the target was an integral part of the life cycle of HIV, the protein CCR5 on immune CD4 + T cells – a current target in clinical trials aimed at changing a person's immune system against HIV is resistant. The modified tracrRNA improved cutting at this site and the inactivation of CCR5, which will hopefully result in better protection of the immune system.

The new design also improved activity at the HBB gene and at the BCL11A site, both of which are sickle cell diseases The results could eventually be new or improved gene therapies, "Scott said, adding that his team is at the beginning of this long scientific process ,


A new CRISPR-Cas9 protein to increase the precision of gene processing


Further information:
Tristan Scott et al., Improved Cas9 Activity by Specific Modifications of TracrRNA, Scientific Reports (2019). DOI: 10.1038 / s41598-019-52616-5

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City of Hope

Quote :
Modified CRISPR Gene Editing Tool May Improve Therapies for HIV and Sickle Cell Disease (2019, November 7)
retrieved on November 8, 2019
from https://phys.org/news/2019-11-crispr-gene-tool-therapies-hiv.html

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