GENETIC CONSTRUCT PMITOASCPF1, CODING NUCLEASE ASCPF1 WITH DETERMINANT OF IMPLANTATION IN THE MITOCHONDRIA OF HUMAN CELLS

摘要

FIELD: biotechnology.;SUBSTANCE: invention relates to biotechnology, medicine and treatment in hereditary diseases. Proposed is a genetic construct pMitoAsCpfl that provides the expression of nuclease AsCpfl specifically implanted in the mitochondria of human cells. This construct comprises a cytomegalovirus promoter, a mitochondrial gene leader sequence COX8A, 3xFLAG, nuclease AsCpfl with SEQ ID NO:1, T2A, TurboGFP gene, 3’-UTR of SOD2 gene, origin of replication, ampicillin resistance gene.;EFFECT: invention can be used to eliminate mitochondrial DNA containing pathogenic mutations associated with hereditary mitochondrial pathologies.;1 cl, 1 dwg