Therapeutic approaches of the CRISPR/Cas genome editing system for genetic diseases in humans and model animals
https://doi.org/10.25557/2073-7998.2018.05.3-12
Abstract
Since the opening of the CRISPR / Cas genome editing system, this technology has made significant progress thanks to numerous modifications and improvements, coming close to application in clinical medicine. At present, the issue on its use for the treatment of genetic diseases, where the gene causing the disease is known, is on the agenda. With the help of this system, it is supposed to correct the mutant gene in cells taken from the patients themselves with the subsequent introduction of these cells back to the patient, or to correct in situ the mutation directly on the spot in the target gene in patients.
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Review
For citations:
Mglinets V.A. Therapeutic approaches of the CRISPR/Cas genome editing system for genetic diseases in humans and model animals. Medical Genetics. 2018;17(5):3-12. (In Russ.) https://doi.org/10.25557/2073-7998.2018.05.3-12
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