Achievements in molecular biology: CRISPR/Cas9 in gene therapy (literature review)

Oleksii Voroshchuk; Olha Yaremchuk

CRISPR/Cas9 provides high precision and efficiency in altering genetic sequences, therefore significant in gene therapy. Mutations can be corrected, pathological genes can be removed, and the functional ability of proteins can be restored by CRISPR/Cas9. The review aimed to analyse the possibilities of using gene editing technology to treat cancer, genetic, and infectious diseases in the available studies published in 2013-2024. This publication analysed the use of CRISPR/Cas9 in experimental models for treating Duchenne muscular dystrophy, cystic fibrosis, sickle cell anaemia, acquired immunodeficiency syndrome and cancer. In Duchenne muscular dystrophy, genome editing helps increase the level of utrophin, which compensates for the dystrophin deficiency. In cystic fibrosis, CRISPR/Cas9 is used to correct defects in the CFTR gene, and in human immunodeficiency virus therapy, it is used to remove proviral deoxyribonucleic acid from infected cells. However, the technology also has certain limitations, such as the risk of off-target changes in the genome and the difficulty of delivering CRISPR/Cas9 into cells. Therefore, in 2024, CRISPR/Cas9 requires further improvement in clinical practice. CRISPR/Cas9 has great potential to change the approach to the treatment of incurable diseases in the future. The practical value of the study conducted by the authors is the presentation of a ready-made summary of the CRISPR/Cas9 system and a thorough analysis of the results of its use in the treatment of various diseases, which can be used to assess what prospects this technology has for future use

genome editing technologies; adeno-associated viruses; Duchenne muscular dystrophy; cystic fibrosis; haemoglobinopathies; acquired immunodeficiency syndrome; cancer

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