Modulation of Insulin Gene Expression with CRISPR/Cas9-based Transcription Factors

Bakhytzhan Alzhanuly, Zhussipbek Y. Mukhatayev, Dauren M. Botbayev, Yeldar Ashirbekov, Nurlybek D. Katkenov, Nurlan T. Dzhaynakbaev, Kamalidin O. Sharipov
2021 Open Access Macedonian Journal of Medical Sciences  
The discovery and use of CRISPR/Cas9 technology have enabled researchers throughout the globe to continuously edit genomes for the benefit of science and medicine. Diabetes type I is one field of medicine where CRISPR/Cas9 has a strong potential for cell therapy development. The long-lasting paucity of healthy cells for clinical transplantation into diabetic patients has led to the search of new methods for producing β-cells from other human cell types. Embryonic stem cells are being studied
more » ... ldwide as one most promising solution of this need. Aim: The aim of the study is to to check the feasibility of modulating human insulin transcription using CRISPR/Cas9-based synthetic transcription regulation factors. Results: A new approach for creating potential therapeutic donor cells with enhanced and suppressed insulin production based on one of the latest achievements of human genome editing was developed. Both synthetic transcription activator (VP64) and transcription repressor (KRAB) proteins were shown to function adequately well as a part of the whole CRISPR/Cas9-based system. We claim that our results have a lot to offer and can bring light to many studies where numerous labs are struggling on to treat this disease.
doi:10.3889/oamjms.2021.6980 fatcat:po4lkptzszfbjkylkbylozux7q