One of the additional complications of treating CNS diseases is that it is separated from the blood by the blood-brain barrier, which allows the brain to miss all the medicinal substances, even low-molecular. Preparations for gene therapy, which are nucleic acids of various sizes, are even more difficult to deliver to the central nervous system.
Scientists from Oxford University have tried to overcome this complexity with the help of exosomes - synthesized by many cells of the body of microscopic vesicles from the cell membrane, serving for the transport of various, often high-molecular chemical compounds.
In the experiment, the researchers obtained exosomes from the immune cells of mice and filled them with small interfering RNAs (siRNA) capable of deactivating the BACE1 gene. The product of this gene - beta-secretase enzyme 1 - plays a key role in the development of Alzheimer's disease.
Then, a membrane of siRNA-loaded exosomes was loaded with a rabies virus protein, which ensures its binding to brain cholinoretseptors. This protein was supposed to "direct" the exosomes to the site of action.
After introducing their own exosomes prepared in this way back into the body of mice, scientists noted a 60 percent decrease in the activity of the target gene, which indicates the effectiveness of the method. According to the study's head Matthew Wood (Matthew Wood), this natural transport system was used to carry medicines for the first time.
As the researchers and independent experts noted, the developed technique is quite universal and can be used to treat many diseases.
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