Artificial chromosomes help to cope with hereditary diseases
Last reviewed: 23.04.2024
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According to the press service of the Institute of Stem Cells, scientists from the Center for the Construction of Chromosomes, located at the University of Tottori in Japan, managed to get artificial human chromosomes that can be used for gene or cell therapy, in order to get rid of hereditary diseases.
Professor Mitsuo Oshimura, who is the Director of the Center, has been conducting serious research in the field of treating hereditary diseases for many years by introducing artificial chromosomes called so-called induced pluripotent stem cells, which are formed from developed somatic cells by using the expression method of a set of four genes - transcription factors).
The researcher came to the conclusion that the technique he proposed makes it possible to treat diseases like Duchesne's myodystrophy - a dangerous disease of the neuromuscular apparatus, in which changes in muscle fibers occur. The reason for this disease is considered a mutation of the gene associated with the synthesis of a special protein - dystrophin. And the symptoms are noticeable already in the first years of life and pose a serious threat to health.
Professor Oshimura put his experiments on mice, since this method of obtaining actual evidence on the effectiveness of certain treatments, pharmacological drugs and medical equipment is most suitable for qualitative research and does not require additional equipment, such as a facility for medical waste disposal, a large number of diagnostic equipment and means of monitoring the condition of patients.
As a result of the experiments, it was confirmed that gene therapy, based on the use of artificial chromosomes, actively promotes the normalization of the work of muscle tissue in mice. The meaning of the new technique is the construction of a chromosome, which must carry the desired DNA fragment in a "corrected" form - without mutation. Next, the chromosome is placed in a prepared stem cell that acts as a transport for the "right" gene. Then, during the cultivation process, new cells are obtained that can be transplanted into diseased organs or tissues.
Experts believe that the new technology has a great future, because by applying it, large sections of DNA can be inserted into the cells without fear for the integrity of the existing genome. The advantages of chromosomes created artificially, over viral or other vector systems are enormous genetic capacity, stability at the mitotic level, the absence of threats to the host genome, and the possibility of withdrawing modified chromosomes from cells.
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