CRISPR/Cas SYSTEM FOR GENOME EDITING IN PLURIPOTENT STEM CELLS
E.A. Vasilieva,1,* G. Melino,2,3 N.A. Barlev 1,2
1 Institute of Cytology RAS, 2 Technological University, St. Petersburg,
and 3 MRC Toxicology Unit, Leicester, LE1 9HN, United Kingdom;
* e-mail: slkd-k@mail.ru
Genome editing systems based on site-specific nucleases became very popular for genome editing in modern
bioengineering. Human pluripotent stem cells provide a unique platform for genes function study, disease
modeling, and drugs testing. Consequently, technology for fast, accurate and well controlled genome manipulation
is required. CRISPR/Cas (clustered regularly interspaced short palindromic repeat/CRISPR-associated)
system could be employed for these purposes. This system is based on site-specific programmable nuclease
Cas9. Numerous advantages of the CRISPR/Cas system and its successful application to human stem cells provide
wide opportunities for genome therapy and regeneration medicine. In this publication, we describe and
compare the main genome editing systems based on site-specific programmable nucleases and discuss opportunities
and perspectives of the CRISPR/Cas system for application to pluripotent stem cells.
Key words:
CRISPR/Cas, genome editing, pluripotent stem cells, ZFNs, TALENs
| Back
| Contents
| Main |
|
