LIPID NANOPARTICLES HELPED TO CURE GENETIC DISORDERS
25.08.2020
Genetic disruptions lead to very serious consequences. For example, the production of proteins responsible for blood clotting or the removal of cholesterol from the bloodstream may be disrupted. It is possible to change such violations pointwise by delivering the missing genetic molecules. However, up to now systems of this kind have not been created that are safe for the body.
Researchers have previously created several types of spherical lipid-like nanoparticles for messenger RNA. Scientists have now tested these systems for delivering RNA to the cells of animals with genetic abnormalities. The authors carried out experiments with different types of particles, modifying their structure. As a result, the authors found the most optimal nanoparticle in terms of structure and stability, which can efficiently deliver messenger RNA to an address.
Tests in mice have shown that such lipid nanostructures can indeed treat genetic pathologies. In one experiment, scientists injected nanoparticles containing messenger RNA with instructions to produce human clotting factor VIII into the bloodstream of normal mice and mice with hemophilia. Deficiency of this protein causes a bleeding disorder. Within 12 hours, mice with a deficiency produced so much human clotting factor VIII that it reached 90% of normal. Organ examinations in both protein-deficient and normal mice showed that the treatment did not cause damage.
In the second experiment, the researchers inserted two types of instructions into the nanoparticles: messenger RNA, which carries the genetic code for the DNA base editor, and guide RNA, to make sure that changes take place in the PCSK9 gene in the liver. It is known that dozens of mutations that increase the activity of this gene increase cholesterol levels, reducing the rate at which this substance is released from the bloodstream. The results showed that the therapy helped change 60% of the target base pairs in the PCSK9 gene - a fairly high figure, which is enough to normalize the body's condition. Scientists also found that a very low dose of the drug was needed to obtain such an effect.
Researchers have previously created several types of spherical lipid-like nanoparticles for messenger RNA. Scientists have now tested these systems for delivering RNA to the cells of animals with genetic abnormalities. The authors carried out experiments with different types of particles, modifying their structure. As a result, the authors found the most optimal nanoparticle in terms of structure and stability, which can efficiently deliver messenger RNA to an address.
Tests in mice have shown that such lipid nanostructures can indeed treat genetic pathologies. In one experiment, scientists injected nanoparticles containing messenger RNA with instructions to produce human clotting factor VIII into the bloodstream of normal mice and mice with hemophilia. Deficiency of this protein causes a bleeding disorder. Within 12 hours, mice with a deficiency produced so much human clotting factor VIII that it reached 90% of normal. Organ examinations in both protein-deficient and normal mice showed that the treatment did not cause damage.
In the second experiment, the researchers inserted two types of instructions into the nanoparticles: messenger RNA, which carries the genetic code for the DNA base editor, and guide RNA, to make sure that changes take place in the PCSK9 gene in the liver. It is known that dozens of mutations that increase the activity of this gene increase cholesterol levels, reducing the rate at which this substance is released from the bloodstream. The results showed that the therapy helped change 60% of the target base pairs in the PCSK9 gene - a fairly high figure, which is enough to normalize the body's condition. Scientists also found that a very low dose of the drug was needed to obtain such an effect.
Thanks for subscribing!
