MITOCHONDRIAL TARGETING AND IMPORT OF A VIRUS TO DELIVER A NUCLEIC ACID

摘要

Compositions and methods for introducing nucleic acid molecules into mitochondria are described. The compositions comprise at least one virion comprising: i) a recombinant viral vector comprising a human mitochondrial gene operably linked to a mitochondrial promoter, and ii) a viral capsid comprising a mitochondrial targeting sequence, and permit direct delivery of the nucleic acid molecules into the mitochondria. The use of such compositions for the delivery and expression of a wild-type mitochondrial gene in mitochondria, and for the treatment of mitochondrial related diseases, is also described.