Isolated mitochondria as delivery vehicles and stimulators of protein synthesis

Abstract

The present invention relates generally to isolated mitochondria derived from mtDNA-depleted cells (Rho0-mito) that are advantageously used as delivery vehicles for heterogenous proteins or nucleic acids or chemical agents. In some embodiments, proteins or nucleic acids or chemical agents are introduced into a cell, transported into, transferred or expressed in the mitochondria, thereafter or before mtDNA is removed and mtDNA-free mitochondria are extracted from the cell. These mitochondria are used for therapeutic treatment of mitochondria-related diseases such as maple syrup syndrome, citrin deficiency, Nieman-Pick disease, X-linked adrenoleukodystrophy (X-ALD), Pyruvate Dehydrogenase Deficiency, respiratory chain diseases, mutations in mitochondrial respiratory chain proteins, fatty acid metabolism disorders, or cancer with reduced likelihood of causing an immunogenic response. Additionally, in some embodiments the present invention relates to isolated mitochondria, including but not necessarily isolated mitochondria derived from mtDNA-depleted cells (Rho0-mito), that are exogenously introduced into cells to enhance production of endogenous or heterogenous proteins.

Dependencies are inferred from the claim text.