New Nanoparticles Deliver Gene Editing Tool with Higher Efficiency
Nanoparticles improve the CRISPR/Cas9 tool
Gene editing is a group of technologies that allows scientists to modify the DNA of an organism, add, remove or modify genetic material at specific locations in the genome.
CRISPR/Cas9 has attracted much attention as a major gene publishing tool in recent years, which is promising for the treatment and prevention of diseases such as some monogenic diseases and more complex diseases such as cancer.
However, CRISPR/Cas9 is a large molecular complex, including the Cas9 protein which is capable of cutting the double strands of the targeted genomic sequence and a modified RNA sequence allowing Cas9 to find its target.
This large molecular complex makes it difficult to deliver directly into the cell nucleus, which limits its power and use. In this new study, researchers from the Institute of Chemistry, the Chinese Academy of Sciences and Tufts University in the United States developed a type of biodegradable synthetic lipid nanoparticle to improve CRISPR/Cas9.
These nanoparticles can penetrate more easily into a cell
According to the study published in the journal Advanced Materials, these nanoparticles can penetrate more easily into the cell, which contains the messenger RNA encoding Cas9. When these nanoparticles enter the cell, they quickly disassemble and release their contents into the cell.
Then, the cell will take over to manufacture the Cas9 protein from the messenger RNA matrix released by the nanoparticles.
In tests on mice, researchers tried to reduce a gene encoding the PCSK9 protein, the loss of which can lower bad cholesterol levels and reduce the risk of cardiovascular disease.
After using nanoparticles to deliver CRISPR/Case9, the expression of PCSK9 was reduced by 80% in the liver.
These nanoparticles are one of the most effective CRISPR/Case9 carriers ever seen
The researchers stated that these lipid nanoparticles were one of the most effective CRISPR/Case9 carriers they have ever seen for therapeutic applications of the gene editing tool.
