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Genome editing using the CRISPR/Cas9 system and its application in haematology


Authors: M. Racková 1,2;  V. Bakardjieva-Mihaylova 1,2;  M. Slámová 1,2;  L. Boublíková 1,2;  K. Škvárová Kramarzová 1,2
Authors‘ workplace: CLIP – Childhood Leukaemia Investigation Prague 1;  Klinika dětské hematologie a onkologie, 2. lékařská fakulta, Univerzita Karlova a FN Motol, Praha 2
Published in: Transfuze Hematol. dnes,1, 2019, No. Online only 1, p. 1-19.
Category:

Overview

Genome editing is considered to be the biggest advance in molecular genetics since the discovery of the polymerase chain reaction. This method enables the introduction of changes in a target DNA sequence with unprecedented accuracy and efficiency. Since it greatly facilitates the study of genes and biological processes, it has been employed in basic research across all biotechnology fields. The CRISPR/Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR Associated Protein 9) system is the most widely used genome-editing platform. Due to its relatively easy construction and high efficiency, this system has revolutionized the field of molecular biology and also holds enormous potential in translational medicine. The aim of this review is to introduce genome-editing technique and summarize the most important applications of this unique technology in research, diagnostics and treatment of haematologic diseases.

Keywords:

genome editing – CRISPR/Cas9 – haematology – monogenic disorders


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