MOSCOW, August 14. Scientists from the Institute of Cytology and Genetics of the Siberian Branch of the Russian Academy of Sciences have created a new method for testing medicinal compounds. According to the authors, the developed technique allows one to quickly and without additional experiments evaluate the effect of substances on neurons in people with Parkinson's and Huntington's diseases. The results are published in the journal Biomedicines.
To determine how well a particular drug will suit patients, preliminary drug trials are first carried out on laboratory animals. However, such tests often cannot predict a person's exact reaction to chemicals due to biological differences between humans and animals, explained the Institute of Cytology and Genetics of the Siberian Branch of the Russian Academy of Sciences.
To solve this problem, the researchers created a collection of cell models of neurodegenerative diseases (Parkinson's and Huntington's diseases). They are induced pluripotent stem cells (iPSCs), the experts noted.
To create iPSCs, the patient's blood cells are «reprogrammed» in such a way that they acquire the characteristics of embryonic stem cells, according to scientists. By influencing them, specialists obtain mature cells of any type, in particular neurons.
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"Over the past year, we have improved these models. «We introduced a transgene (a DNA fragment transferred to the genome of an organism to modify its properties) into iPSC cells obtained from patients with Parkinson's and Huntington's diseases, from which special protein molecules called biosensors are synthesized,» said one of the study's authors, senior researcher at the Institute of Cytology and Genetics SB RAS Anastasia Malakhova.
She added that the biosensors are based on fluorescent proteins, by the nature of their glow, scientists can draw conclusions about the state of the cell. This allows them to select suitable drugs without additional experiments.
«After treating a cell with any substance, we immediately see its reaction by the glow of the biosensors. Now drug screening has become faster and more convenient,» Malakhova noted.
Now the specialists are faced with the task of studying the effect of PARP-1 enzyme inhibitors, which are used to treat cancer, on cell models. Scientists suggest that they can be used to preserve brain neurons in neurodegenerative diseases.
The study was conducted with the financial support of the Russian Science Foundation. The results were presented at the 14th International Multiconference «Bioinformatics of Genome Regulation and Structure/Systems Biology» (BGRS/SB2024).