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
| Title |
Year |
Author |
Number |
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|
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2016 |
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|
RU2727540C1 |
