ALEXANDRIA, Va., Oct. 6 -- United States Patent no. 12,753,419, issued on Oct. 6, was assigned to The Trustees of the University of Pennsylvania (Philadelphia).
"Compositions useful for treatment of Charcot-Marie-Tooth disease" was invented by James M. Wilson (Philadelphia), Christian Hinderer (Baltimore) and Eileen Workman (Philadelphia).
According to the abstract* released by the U.S. Patent & Trademark Office: "Provided herein are rAAV and other vectors and compositions useful for treating a patient having CMT2 comprising: (a) a recombinant nucleic acid sequence encoding an engineered human mitofusin 2 coding sequence operably linked to regulatory sequences which direct expression thereof in a human target cell. Also provided are rAAV ...