Compositions and methods for reducing sarcolipin expression and preventing and treating muscular dystrophy and cardiomyopathy

摘要

To inhibit sarcolipin (SLN) expression or activity in cells, as well as in subjects (eg, human patients having or predisposed to have Duchenne muscular dystrophy (DMD)), and in some embodiments further Compositions, recombinant viruses, and recombinant viral vectors for preventing or treating cardiomyopathy comprise a therapeutically effective amount of a SLN inhibitor. Methods for using these compositions, recombinant viruses, and recombinant viral vectors are also described herein. These compositions, recombinant viruses, and recombinant viral vectors and methods of use provide new therapies for DMD and related cardiomyopathy based on reduced expression and / or activity of SLN.