Mitochondrial genome replenishment
    11.
    发明专利

    公开(公告)号:AU2002330938A1

    公开(公告)日:2003-02-17

    申请号:AU2002330938

    申请日:2002-07-31

    Abstract: The invention provides composition and methods for delivering a composition according to the invention comprising a wild-type (wt) mitochondrial DNA (mtDNA) molecule to a mammalian cell. The wt-mtDNA molecule is a functional mtDNA molecule that includes the entire mitochondrial genome. The wt-mtDNA molecule in the composition of the invention has a mitochondrial leader sequence (MLS) peptide attached to facilitate the uptake of the mtDNA molecule into a mitochondrion of the mammalian cell. The delivery of the wt-mtDNA/MLS peptide complex is used as a general replenishment therapy for conditions attributed to mtDNA defects, independent of the specific defect in the mtDNA.

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