13 June 2019

Antisense therapy

Innovative therapy of Parkinson's disease will block the synthesis of alpha-synuclein

"Attending Physician"

Parkinson's disease is a chronic steadily progressive neurodegenerative disease that leads to the development of tremors, impaired musculature and decreased mobility. Pathophysiologically, the disease is associated with the destruction of dopaminergic neurons in the substantia nigra of the brain due to the accumulation of alpha-synuclein protein in them.

It has been shown that in hereditary forms of Parkinson's disease, the number of copies of the gene responsible for the synthesis of alpha-synuclein increases. These repeats are considered one of the most promising therapeutic targets for the hereditary form of the disease. A group of scientists from Osaka University in Japan took up the development of a technique that would work at this level. They published the results of their work in a recent issue of the journal Nature Scientific Reports (Uehara et al., Amido-bridged nucleic acid (AmNA)-modified antisense oligonucleotides targeting α-synuclein as a novel therapy for Parkinson's disease).

During the experiment, scientists synthesized a "mirror" nucleotide sequence corresponding to the DNA site on which alpha-synuclein is synthesized. This sequence, after stabilization, was called an "antisense oligonucleotide". It was assumed that it would be able to block the synthesis of alpha-synuclein by combining with matrix RNA.

In in vitro and in vivo experiments, scientists were able to confirm the effectiveness of antisense nucleotides in reducing the level of alpha-synuclein. Thus, it was found that in mice with induced Parkinson's disease, the level of alpha-synuclein decreased by 81% when using antisense oligonucleotides. The medicinal substance was injected directly into the ventricles of the brain.

It is assumed that such antisense oligonucleotides may be effective not only in the treatment of Parkinson's disease, but also in dementia with Levi's corpuscles, since alpha-synuclein serves as their main component.

In the future, Japanese scientists plan to carry out new studies with antisense oligonucleotides, including with the participation of people suffering from Parkinson's disease.

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