Abstract
Significant progress has been made in the development of genetic technologies in recent decades. Currently, high-performance sequencing and, most importantly, genome editing technologies are widely used and available for laboratories in Russia. Existing technologies are not without drawbacks that significantly hinder further development, in addition, all the necessary reagents and materials, as well as equipment, are produced exclusively abroad. The review highlights the international experience of using genome editing technologies for the treatment and prevention of genetic diseases, vector-borne and viral infections, it offers recommendations for the development of this area in the Russian Federation. Attention is drawn to the legal and ethical regulation, mainly at the level of basic principles. The conclusion is formulated on the need for accelerated adaptation of basic ethical and legal principles for genome editing activities in scientific biomedical activities.
Highlights
Significant progress has been made in the development of genetic technologies in recent decades
Research aimed at achieving this goal has been going on for decades — from triplex-forming oligonucleotides to peptide nucleic acids and polyimines (Eid, Alshareef and Mahfouz, 2018; Koonin, Makarova and Zhang, 2017; Lee et al, 2018), but the development of the platform with adequate specificity and efficiency is still far away. It seems that new methods of genome editing will be discovered through research into natural processes rather than through optimization of CRISPR techniques
TAL nucleases have been used to enhance the efficacy of CAR-T cell therapy (Lucibello, Menegatti and Menger, 2020); in addition, two studies using CRISPR/Cas9 have been approved for this purpose
Summary
The target of using genome-editing nucleases, is only producing double-stranded breaks of chromosomal DNA. Most somatic cells in higher eukaryotes start the NHEJ process with the concomitant occurrence of insertions and deletions more often than copy sequences from the donor DNA provided This is acceptable if the purpose of editing is to knock out a gene or a complex of genes, but it significantly limits the possibility of introducing required nucleotide sequences. Several recent publications report that small molecule inhibitors of key enzymes in the NHEJ process can be effective, but more research is needed to create more reliable reagents (Bischoff, Wimberger, Maresca and Brakebusch, 2020; Li et al, 2017) Another way of increasing the efficiency of insertion of the target gene is the modification of the donor DNA molecule, linking the donor sequence with the guide RNA, and using natural mechanisms for inserting the. The priority of the patient’s interests is established as a principle and in Art. 4 of the Federal Law No 323-FZ dated 21.11.2011 “On the fundamentals of the public health protection in the Russian Federation”
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