City of Hope researchers may have found a way to sharpen the fastest, cheapest and most accurate gene editing method, CRISPR-Cas9, so that it can more effectively cut off unwanted – Desirable genetic information.
This enhanced cutting ability may one day quickly monitor potential therapies for HIV, cell disease and, potentially, other conditions of immunity. a butter knife compared to slicing it with a steak knife, "said Tristan Scott, Ph.D., lead author of the study and a staff research scientist at City of Hope & # 39; s. Center for Gene Therapy. "Other scientists have tried to improve CRISPR disruption through chemical modifications, but that is an expensive process and is like a co-coating blade. Instead, we designed a better pair of scissors that you can buy at any convenience store. "
study, published in Scientific Reports on November 6, was the first time scientists have been systematically passed through RNA sequence guidance to modify it and improve CRISPR-Cas9 technology, Scott said .The Kevin Morris Lab at the City of Hope has filed a patent application saying improved CRISPR-Cas9 design, which may result in a duplication of activity but the exact value depends on the target site, Scott said.
This study is one of the City's many ways Hope, a well-known independent research and treatment center for cancer, diabetes and other life-threatening diseases, helps enable precision medicine. repair foundation blocks that provide accompanying gene therapies with better tools.
Fluid effects may be more "clean" results in cell and mouse experiments aimed at developing new therapies because the target "knocked out" is more successfully removed. More pronounced results can accelerate new therapies from the laboratory to the patients' bedides. In theory, the therapeutic product should have a more successful reduction, which may translate into improved therapy, but further research is needed. The exact mechanics of why this change in the CRISPR system improves gene editing still needs to be determined.
Researchers experimented with cells by making changes to the "trans-activating CRISPR RNA" (also known as "tracrRNA"), which is derived from Streptococcus pyogenes bacteria and a component of components used to guide the genetic scissors (Cas9) in the correct gene sequence. Streptococcus pyogenes Cas9 is the most commonly used genetic scissors. Scientists use an RNA protein system because it provides an explosion of activity that is lost almost 12 hours after being introduced into the cell, which means there is a reduced chance that the human genome will be accidentally edited later , after the "fix" was made, Scott said.
They found that modified tracrRNAs improved the silencing of certain genes by increasing favorable mutations in the genetic material. In this study, the target is an essential component of the HIV lifecycle, the protein CCR5 in immune CD4 + T-cells – a current target in clinical trials aimed at re-engineering a person's immune system to be resistant of HIV. The modified tracrRNA enhances the cleavage at this site and is inactive by CCR5, and is expected to translate it into better protection for the immune system.
The new design also improved the activity of the HBB gene and the BCL11A site, both of which are linked to disease-causing cells and are targeted to generate therapies for current blood-borne diseases of severe illness and premature death.
"If this line of research remains consistent and we can be proud to sharpen the genetic scissors, the result could be new or improved genetic therapies," said Scott, adding that his the team is at the beginning of a long scientific process.
A new CRISPR-Cas9 protein to increase gene editing accuracy
Tristan Scott et al, Improved Cas9 activity by tracrRNA-specific modifications, Scientific standards (2019). DOI: 10.1038 / s41598-019-52616-5
City of Hope
CRISPR's modified gene editing tool can improve therapies for HIV, cell disease (2019, November 7)
acquired November 8, 2019
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