News & Blogs » Protein News » Nanoparticles Carrying CRISPR Create a More Efficient Delivery Tool
In a new study published in
Nature Biotechnology, scientists developed a method using nanoparticles to deliver the CRISPR
genome editing system to modify genes in mice. This method was able to achieve the best success rate on
record for editing live adult animals with CRISPR.
Using a new nanoparticle non-viral type of delivery method, researchers from MIT were able to edit a disease causing gene in about 80 percent of targeted cells. There was a need to discover a safer and more efficient way to deliver CRISPR components. Therefore, researchers developed a system that was able to deliver Cas9 and the RNA guide using nanoparticles without the need for viruses. The first step was chemically modify the guide RNA to protect it from endogenous enzymes that may degrade it before it can reach its final location. The researchers observed the structure of the complex formed by Cas9 and the guide RNA in order to see which modifications they could make without interfering with their binding. It was observed that as much as 70 percent of the guide RNA can be adjusted without interfering with its binding to Cas9. Researchers than packaged these modified RNA guides into lipid nanoparticles and injected them into mice along with nanoparticles containing Cas9 encoding mRNAs.
The experimental focus was to adjust hepatocytes by knocking out Pcsk9 gene, which regulates cholesterol levels in mice. Dominant familial hypercholesterolemia is a rare disorder that is associated with mutations in human version of this gene and causes high cholesterol. Researchers were able to eliminate this gene in more than 80 percent of liver cells and Pcsk9 protein levels were undetectable in mice. Also, in treated mice, scientists were able to observe a 35 percent drop in total cholesterol levels. Scientists are hopeful this new type of delivery method could become a powerful tool for gene editing a number of disease causing genes within the liver and one day to other organs as well.
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