Application of CRISPR/Cas9 system for developing and studying cellular models of inherited disease

  • Authors: Valetdinova K.R1,2,3
  • Affiliations:
    1. Federal Research Center Institute of Cytology and Genetics, the Siberian Branch of the Russian Academy of Sciences
    2. Institute of Chemical Biology and Fundamental Medicine, the Siberian Branch of the Russian Academy of Sciences
    3. State Research Institute of Circulation Pathology, Ministry of Healthcare of the Russian Federation
  • Issue: Vol 11, No 2 (2016)
  • Pages: 10-20
  • Section: Articles
  • URL: https://genescells.ru/2313-1829/article/view/120555
  • DOI: https://doi.org/10.23868/gc120555

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Abstract

Currently, more than 4500 diseases classified as an inherited diseases. Most forms of human inherited diseases are caused by gene mutations. Given to the development of new methods genome editing, particularly with the use of CRISPR/Cas9 system, it is possible to artificially introduce precise mutations in the genome, as well as to correct gene mutations. It is thus possible to create a cellular model of human inherited diseases, as well as to carry out experiments for testing hypotheses and principles of gene and cell therapy. In this review, we consider studies that provide insight into the possibilities of CRISPR/Cas9 system for developing and studying cellular models of human inherited diseases.

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About the authors

K. R Valetdinova

Federal Research Center Institute of Cytology and Genetics, the Siberian Branch of the Russian Academy of Sciences; Institute of Chemical Biology and Fundamental Medicine, the Siberian Branch of the Russian Academy of Sciences; State Research Institute of Circulation Pathology, Ministry of Healthcare of the Russian Federation

Email: valetdinova@bionet.nsc.ru
Novosibirsk, Russia

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