Modern genome editing genetic engineering technologies

Medical Review (Med. pregled), 2025, 61(4), 64-72.

T. Vekov1, L. Dimova2, E. Becheva-Kraychir2, V. Belcheva3

1 Bulgarian Cardiology Institute – Sofia
2 Heart and Brain Hospital – Pleven
3 Trakia University – Stara Zagora

Abstract. Modern genome editing technologies have been the focus of scientific interest over the past two decades. They are the basis of the paradigm for personalized medicine, with thousands of preclinical trials for diagnostic and therapeutic purposes being implemented. However, their clinical application is currently very modest, and the reasons for this are economic and political rather than clinical. The aim of the study is to analyze the genome editing genetic engineering technologies in order to better understand the strategy for the development and application of gene therapies. Many diseases are accompanied by changes in gene expression in vivo, for which solutions based on modern genome editing technologies can be proposed. CRISPR/Cas9 is the most widely used technology in our time for this purpose. The most promising therapeutic applications of CRISPR/Cas9 include the treatment of oncological, hematological, cardiovascular and neurodegenerative diseases. However, there are still some limitations and challenges that need to be addressed in future research. These include issues related to off-target effects, validity, applicabil- ity, chromosomal disorganization and limitations of targeted delivery.

Key words: genetic engineering, technologies, effector nucleases, zinc finger nucleases, CRISPR/Cas9

Address for correspondence: Prof. Toni Vekov, PhD, DSc, e-mail: