Nationwide Children's Hospital, Inc.

Research Institute at Nationwide Children's Hospital

University of California

Jian Liu

Scott Quenton Harper

Lindsay Wallace

Sara Garwick Coppens

Varghese John

Barbara Jagodzinska

Jesus Campagna

Irina Kramerova

Melissa Spencer

Sara Coppens

Sara Garwick

Compositions and methods of use for a compound having a chemical structure of Formula (I) and/or Formula (VII), or a pharmaceutically y acceptable salt or solvate thereof, to increase muscle mass, muscle oxidative capacity, and muscle function and to treat muscular dystrophy, in particular limb girdle muscular dystrophy type 2A/R1/D1 are disclosed.

Compositions and methods for increasing muscle mass and oxidative metabolism

The present invention relates to RNA interference-based methods for inhibiting the expression of the myotilin gene. Recombinant adeno-associated viruses of the invention deliver DNAs encoding microRNAs that knock down the expression of myotilin. The methods have application in the treatment of muscular dystrophies such as Limb Girdle Muscular Dystrophy Type 1A.

Recombinant virus products and methods for inhibition of expression of myotilin

The present invention relates to RNA interference-based methods for inhibiting the expression of the DUX4 gene, a double homeobox gene on human chromosome 4q35. Recombinant adeno-associated viruses of the invention deliver DNAs encoding microRNAs that knock down the expression of DUX4. The methods have application in the treatment of muscular dystrophies such as facioscapulohumeral muscular dystrophy.

Recombinant virus products and methods for inhibition of expression of DUX4

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Los Angeles, CA, United States of America

Jian Liu