Gene editing studies for the treatment of anemia

Author : Fatih Kocabas -Batuhan Mert Kalkan, Gulcin Delal Nozhatzadeh, Fatih Kocabas
Number of pages : 21-29


With the uprising advancements in the genome editing technologies, it is now possible to modify and edit targeted DNA sequences with programmmable endonucleases. The genome editing technologies have become more widely used by researchers after the discovery of zinc finger nucleases (ZFNs) and the transcription activator-like effector nucleases (TALENs) followed by the development of another revolutionary gene editing tool CRISPR-Cas9 system. Improvements in these promising gene editing tools not only reform researchers’ understanding of the human genome but also serve as potential therapeutic approach for inherited blood disorders. The patients who have been suffering from inherited blood disorders are in need of novel therapies as available treatments are limited. Here, in this review, promising new gene editing technologies for the treatment of hemoglobinopathies including β-thalassemia and sickle cell disease are discussed.


HSC, CRISPR/Cas9, gene editing, Anemia, SCD, Thalassemia

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