In Sweden, researchers at Karolinska Institutet have devised a novel technique that ensured the effective delivery of therapeutic proteins and RNA into cells. The method, which has been presented in Nature Communications, has been used to deliver gene editors and therapeutics of proteins.
The method is based on two components: the so-called extracellular vesicles and tiny bubbles, which are secreted naturally in cells. They transport molecules that are biologically active in cells. Two key components have been introduced to improve the quality of these bubbles.
These two key components are as follows: a bacterial protein called intein and a so-called fusogenic protein associated with a virus. The fusogenic protein would help the bubbles combine with the endosomal membrane and then release the contents into the cell. The intein would cut itself and would help release therapeutic proteins present inside the cell.
According to Professor Samir El Andaloussi, who is the last author of the study and researcher at the Department of Laboratory Medicine, Karolinska Institute, the engineering strategy is innovative and it presents an important step ahead in the development of extracellular vesicle technology. It can effectively overcome key barriers, which includes poor endosomal escape and a limited intracellular release.
A diverse range of conditions, such as systemic inflammation, genetic diseases, and neurological disorders have been treated with a method that has the potential of engineered EVs, which is a versatile platform that delivers therapeutics.
Dr. Xiuming Liang is the first author of this study. He has devised this technology that can effectively increase the feasibility of including advanced medicines, which improve the efficiency and reliability of therapeutic delivery in target cells.
Experiments were conducted on cells and live animals. In these studies, Cre recombinase was efficiently delivered in such a way that a protein could be cut and pasted into DNA and the Cas9/sgRNA.
These components were then used to edit genes. Cre recombinase was used to load extracellular vesicles. Then, these vesicles were injected into the brains of mice. This caused a significant change in the cells of hippocampus and cortex brain structures.
In conclusion, there is hope to use the CRISPR/Cas9 gene scissors or similar tools. These tools can treat severe genetic diseases, which occur in the central nervous system. The diseases were Huntington’s disease and spinal muscular atrophy. Systemic inflammation in mice could be treated with this technique.