Novel Therapeutic Approaches to Mitochondrial Disease

J.   A.   Ibdah; R.   M.   Payne

Abstract

Defects in mitochondrial function cause serious pediatric morbidity and mortality. Most mitochondrial disorders are caused by nuclear gene defects. To overcome the limitations of viral vectors for delivering gene products to mitochondria, we used protein transfection domains that cross cell membranes to deliver proteins expressed by nuclear genes to mitochondria.

Keywords: fatty acids, oxidation, ptd, tat, fusion proteins, mitochondria

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