01 February 2016

Reprogrammed cells will soon become publicly available

Cell rejuvenation technology is becoming mass

Julia Rudy, Vesti 

The use of stem cells in medicine is gaining momentum every year. At the same time, the ethical side of this issue related to the production of embryonic stem cells from human embryos is widely discussed.

In 2006, Japanese scientist Shinya Yamanaka developed a technology for genetic reprogramming adult cells into pluripotent stem cells (a kind of multifunctional). They are also called induced pluripotent stem cells. That is, it has become possible to return the cells of the adult organism that have already determined their functions to the "young" embryonic state, avoiding the aforementioned ethical difficulties.

In 2012, Shinya Yamanaka received the Nobel Prize in Medicine and Physiology for this discovery. But this technique has its drawbacks: the scientist and his team used genetic constructs that are in viral vectors to reprogram cells. It is worth noting that this method has one drawback: viral DNA, getting into a cell, is embedded in its genome, and thereby changes it. All this can lead to unpredictable consequences, including malignant transformations, that is, cancer.

In 2015, Merck introduced Simplicon technology, which makes it possible to obtain pluripotent stem cells using RNA molecules instead of DNA for reprogramming. A detailed description of the technology is in the article of the journal Cell Stem Cell (Yoshioka et al., Efficient Generation of Human iPSCs by a Synthetic Self-Replicative RNA).

"RNA, unlike viral DNA, is not embedded in the genome and lives for a very short time, which makes the procedure for obtaining stem cells more accessible, effective, and, most importantly, safer," comments Professor Sergey Lvovich Kiselyov, Doctor of Biological Sciences, Scientific Director of the Epigenetics Department, Chief Researcher at the Vavilov Institute of General Genetics of the Russian Academy of Sciences.

The Simplicon reprogramming system is a synthetic RNA strand encoding proteins responsible for the reprogramming process. With the help of this RNA molecule, differentiated ("adult") cells turn into pluripotent (again "young"), and synthetic RNA molecules are completely removed from the cell.

In December 2015, R&D Magazine included Simplicon technology in the list of the 100 best inventions of 2015. The prestigious awards of the R&D Magazine 100 Award, which are also called "Oscars of Inventions", are received by the creators of the 100 most innovative developments in the field of science and technology that have entered the market for the year.

Why do I need to reprogram cells?Reprogramming of cells is necessary in order to obtain induced pluripotent stem cells, which can then be used to grow any specialized tissue of the body.

For example, Yamanaki's team subsequently received retinal cells, which were then implanted into elderly people. It is expected that the use of "rejuvenated" cells will restore the vision of the elderly (there are already positive results).

Pluripotent stem cells can even produce neurons that synthesize the hormones serotonin or dopamine. Currently, the possibilities of using the cell transplantation method for the treatment of diabetes, as well as some neurodegenerative diseases, are being actively investigated. In the near future, clinical trials of methods of treatment with "young" cells of cardiovascular diseases should begin.

When will cell reprogramming technology become massively available?Clinical trials on the use of pluripotent stem cells began relatively recently, and normally they take 10-12 years.

The first implantation of retinal cells in clinical trials took place in 2013. But in a dozen years, in case of a successful outcome of the trials, the use of induced pluripotent stem cells for transplants will become an integral element of medicine.

In parallel with the development of cell transplantation methods, the iPS cells produced today already serve for the personal selection of medicines (previously mice were used for these purposes at best). Stem cells are also used to elucidate the mechanisms of disease development.

For example, it is known that many medications have cardiotoxicity, that is, their use harms a person's heart. The use of reprogramming technology makes it possible to obtain heart muscle cells and cardiomyocytes from the patient's skin, test the drug on them and, in case of a positive response, recommend it to the patient. Thus, today reprogramming technology is already used in personalized medicine and allows you to select medications more accurately.

Cell Reprogramming in RussiaRussian scientists are also engaged in research and development on cell reprogramming.

The greatest success in this direction was achieved by the staff of the Department of Genetic Foundations of Cellular Technologies of the Vavilov Institute of General Genetics of the Russian Academy of Sciences: in 2009, he managed to patent his method of reprogramming using RNA.

New technologies like Simplicon provide the convenience of research and reduce their cost, allowing more scientists to engage in this area. Professor Sergey Lvovich Kiselyov notes the following achievements of Russian scientists: "At the moment we are studying the application of the method of reprogramming using RNA in various fields of medicine. For example, in the field of neurology, we were able to study the effectiveness of using pluripotent stem cells for the treatment of Parkinson's disease. In addition, we are exploring the possibilities of stem cells in the treatment of cardiovascular diseases, and we also assume that we will be able to use reprogrammed cells to treat genetically inherited pathologies related to vision."

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01.02.2015
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