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Defects in mitochondrial function are responsible for many human diseases. Two major problems have hindered gene therapy of mitochondria defects: 1) Mitochondria are in all tissues of the body so a gene therapy must be capable of reaching all tissues. 2) The therapy must localize specifically to mitochondria. Using a short peptide sequence derived from the HIV genome, called TAT, the hypothesis that TAT fusion proteins could cross both mitochondrial membranes and incorporation of a mitochondrial signal sequence (MTS) into a TAT fusion protein would allow processing and localization of…mehr

Produktbeschreibung
Defects in mitochondrial function are responsible for many human diseases. Two major problems have hindered gene therapy of mitochondria defects: 1) Mitochondria are in all tissues of the body so a gene therapy must be capable of reaching all tissues. 2) The therapy must localize specifically to mitochondria. Using a short peptide sequence derived from the HIV genome, called TAT, the hypothesis that TAT fusion proteins could cross both mitochondrial membranes and incorporation of a mitochondrial signal sequence (MTS) into a TAT fusion protein would allow processing and localization of exogenous proteins in mitochondria was tested. Using isolated mitochondria, cultured cells, animals, and model membranes, TAT-MTS proteins rapidly transduces into large unilamellar vesicles, cells and mitochondria, and persisted over time in vitro and in vivo. TAT-MTS fusion proteins will be a useful tool in understanding mitochondrial function as well as the development of mitochondrial protein therapies.
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Autorenporträt
Victoria Del Gaizo Moore received her PhD in Molecular Medicine from Wake Forest University School of Medicine in 2004. She then completed a postdoctoral fellowship with Anthony Letai, MD, PhD at Dana-Farber Cancer Institute, Harvard Medical School in 2009. Currently she is an Assistant Professor in the Chemistry Department at Elon University.