Experts of the Genome Editing Laboratory at RCMG Prepared an Overview of the Latest Scientific Articles on Improving the Delivery Efficiency of CRISPR-Cas Components

The key step in vivo genome editing (for the treatment of human diseases) is the direct delivery of artificial nucleases and donor DNA molecules to somatic cells. It is not possible to control the dosage of artificial nucleases, and this factor, among others, reduces the overall efficiency of in vivo genome editing.

Experts of the genome editing laboratory at RCMG gave an expert assessment of the latest research in the field of improving the delivery efficiency of CRISPR-Cas components, evaluated the possibility of using non-viral delivery methods for clinical use. Delivery of CRISPR-Cas with the protein Cas + sgRNA (ribonucleoprotein complex, RNP complex) has shown its effectiveness. Another its advantage is the reduction of non-specific activity. The article was published in the journal Molecular Biotechnology (Q3). A total of one hundred and twenty one scientific publications are included in the review. This is the first complete literature review dedicated to the delivery of a new form of genome editing Cas + sgRNA.

- We have seen that there is none of  CRISPR-Cas components delivery methods, among the wide variety, that would meet all our requirements. Today, there is not enough data to distinguish a suitable method applicable in human being. The goal of our laboratory is to develop new methods of treatment of hereditary human diseases, and we continue our search as well as keep up with the experience of colleagues, - said Svetlana Smirnikhina, the Head of the laboratory.