Perspectives and limitations of karyotype editing and chromosome therapy
https://doi.org/10.25557/2073-7998.2018.10.35-37
Abstract
Chromosome diseases are genetically determined diseases, manifested by a variety of symptoms. Despite the high frequency of chromosome abnormalities and their clinical significance, there are no approaches to effective treatment of patients and to the correction of large chromosome defects. Several methods for the eliminating of the mutant chromosome from the cell are proposed: through spontaneous loss of the ring chromosome during the culturing of induced pluripotent stem cells (iPSC) and by removing its centromere using the CRISPR/Cas9 system. It was shown that the ring chromosome can be created using CRISPR/Cas9 technology. We tested the first approach for ring chromosome elimination and showed that during the culturing of iPSCs the ring chromosome 13 formed stable fragments, while the ring chromosome 22 was transmitted unchanged. Obviously, the loss of the ring chromosome in iPSCs is not strictly a universal process, and the technologies that can form the basis of chromosome therapy require considerable improvement.
About the Authors
A. A. Kashevarova
Research Institute of Medical Genetics, Tomsk National Research Medical Center, Russian Academy of Sciences, Tomsk
Russian Federation
Russian Federation
O. L. Serov
Institute of Cytology and Genetics, Russian Academy of Sciences, Siberian Branch, Novosibirsk
Russian Federation
Russian Federation
I. N. Lebedev
Research Institute of Medical Genetics, Tomsk National Research Medical Center, Russian Academy of Sciences, Tomsk
Russian Federation
Russian Federation
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Review
For citations:
Kashevarova A.A., Serov O.L., Lebedev I.N. Perspectives and limitations of karyotype editing and chromosome therapy. Medical Genetics. 2018;17(10):35-37. (In Russ.) https://doi.org/10.25557/2073-7998.2018.10.35-37
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