Protein replacement therapy for mitochondrial disorders

M. Rapoport, D. Marcus, A. Saada, T. Erlich, R. Hadad, H. Greif, M. Lichtenstein, H. Lorberboum-Galski, Mitochondrion, Volume 24, Supplement, September 2015, Page S35, ISSN 1567-7249, doi:10.1016/j.mito.2015.07.096.

The approach is to fuse the Mitochondrial targeting Sequence (MTS), with the delivery peptide TAT [HIV-transactivator of transcription (TAT) peptide]. This novel approach has been tested using different mitochondrial proteins implicated in mitochondrial human diseases: Lipoamide Dehydrogenase (LAB), C6ORF66 and Frataxin, and have been evaluated in vitro, in patients' cells and in vivo, in mouse models. In patient's cells and in mice tissues, including the brain, AT-MTS-Mitochondrial fusion proteins arrive at the cells and their mitochondria rapidly and efficiently, getting an improvement of the mitochondrial functions and life span in animal models.


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