MODIFICATION OF STRUCTURE OF SpCas9 NUCLEIC ACID GUIDING RNA MOLECULE TO ENSURE IMPORT OF HUMAN CELLS INTO MITOCHONDRIA Russian patent published in 2020 - IPC A61K48/00 C12N15/00 

FIELD: biotechnologies.

SUBSTANCE: invention relates to the biotechnology. Disclosed is a mitochondria imported RNA with subdomains of transport RNA lysine HD and HF. Each of the presented RNA guides (gRNA-HF-HF, gRNA-HF-HD, gRNA-HD-HF and gRNA-HD-HD) interacts with human mitochondrial tRNA-lysyl synthase (pre-KARS2) and ENO2 protein, which are imported into mitochondria together with RNA guide. In the mitochondria, the RNA guide binds to SpCas9 nuclease, forming a functional complex which introduces double-strand breaks in mtDNA, which lead to its elimination by enzymes of the mitochondrial replication system. Total mtDNA pool in mitochondria is recovered by remaining mtDNA.

EFFECT: providing a system for eliminating certain haplotypes of RGEN/SpCas9 mtDNA and a method for delivering RNA thereof in parts of organelle.

1 cl, 2 dwg, 1 ex