Thursday, May 10, 2012
A University of Miami team has
used mitochondrion-targeted gene therapy to restore functional NADH dehydrogenase subunit 4 levels in Leber's
hereditary optical neuropathy.1 Next steps include optimizing the
approach to cover several variants of the disease and determining the therapy's
potential advantages over pharmacological strategies in development.
Osherovich, L. SciBX 5(19); doi:10.1038/scibx.2012.484
Published online May 10, 2012
Yu, H. et al. Proc. Natl. Acad. Sci. USA; published online April 20,
Contact: John Guy, University of Miami Miller School of Medicine, Miami,
Beltran, W.A. et al. Proc. Natl. Acad. Sci. USA 109, 2132-2137
Ji, F. et al. Proc. Natl. Acad. Sci. USA; published online April 18,
AND INSTITUTIONS MENTIONED
The Children's Hospital of Philadelphia, Philadelphia, Pa.
Edison Pharmaceuticals Inc., San Jose, Calif.
Santhera Pharmaceuticals Holding AG (SIX:SANN), Liestal, Switzerland
University of Miami, Miami, Fla.
University of Miami Miller School of Medicine, Miami, Fla.
University of Pennsylvania, Philadelphia, Pa.
University of Southern California, Pasadena, Calif.