08 September 2014

Cage, heal yourself!

Sick cells were forced to produce medicine on their own

Asya Gorina, Vesti

In the near future, it will be possible to treat muscular dystrophy with the help of the development of scientists from the Scripps Research Institute in Florida. The team has created a unique technique for transforming diseased cells into production sites for molecules capable of treating severe forms of this disease.

"We use the cell as a reaction vessel, and the pathogenic effect as a catalyst for the synthesis of a natural drug inside the affected cell," explains the lead author of the study, Professor Matthew Disney (Matthew Disney).

According to scientists, the main advantage of the technique is that the necessary chemical compounds acting as a drug are synthesized exclusively in diseased cells, while healthy ones remain untouched. This opens a new chapter in the history of targeted therapy: perhaps in the future it will be possible to treat other localized diseases and even cancer in the same way.

Article by Rzuczek et al. A Toxic RNA Catalyzes the In Cellulo Synthesis of Its Own Inhibitor with a description of a new promising method of therapy was published in the publication Angewandte Chemie.

The technology is based on the observation that small, low-molecular compounds can pass through the hemato-encephalic barrier, while more complex compounds turn out to be too large for this. In the new technique, small molecules not only overcome the obstacle, but also inhibit the wrong process – the basis of the disease. Substances bind to their target inside cells that produce an RNA defect, for example, causing myotonic dystrophy.

Let's take an example. Myotonic dystrophy of the second type is caused by an RNA defect, a tetranucleotide repeat, in which a series of four nucleotides is repeated several times, unlike normal sequences in healthy cells. In this case, the cytosine-cytosine-uracil-guanine repeat interferes with the MBNL1 protein, resulting in RNA splicing anomalies. This is a complex process and leads to the development of the disease.

In an experiment by Disney and his colleagues, a pair of specially designed small molecules-"modules" - binds to neighboring parts of a defect in a living cell, as a result of which these groups are joined together. In such conditions, the parts adjacent to each other are connected together, the RNA defect is deactivated and the disease recedes.

"We were able to confirm this with the example of living cells," says study co–author Suzanne Rzuczek.

The main process used by Disney and his colleagues is called click chemistry: chemicals are quickly produced by attaching small molecules-modules, similar to how it happens in nature naturally.

"In my opinion, our technique is the best possible application of click chemistry in practice," Disney admits in a press release from Scripps Florida Scientists Make Diseased Cells Synthesize Their Own Drug.

Given the predictability of the process and the almost infinite number of possible combinations, the developers believe that a complete transition from standard treatment to this type of targeted therapy can be completely justified. RNA is an ideal target because it is modular, just like compounds-molecular templates.

With the help of this technique, you can cure or stop a lot of severe genetic diseases, for example, amyotrophic lateral sclerosis, Huntington's chorea and many other ailments for which there is currently no suitable treatment.

Portal "Eternal youth" http://vechnayamolodost.ru08.09.2014

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