Long-term enhancement of skeletal muscle mass and strength by single gene administration of myostatin inhibitors

AM Haidet, L Rizo, C Handy… - Proceedings of the …, 2008 - National Acad Sciences
AM Haidet, L Rizo, C Handy, P Umapathi, A Eagle, C Shilling, D Boue, PT Martin, Z Sahenk…
Proceedings of the National Academy of Sciences, 2008National Acad Sciences
Increasing the size and strength of muscles represents a promising therapeutic strategy for
musculoskeletal disorders, and interest has focused on myostatin, a negative regulator of
muscle growth. Various myostatin inhibitor approaches have been identified and tested in
models of muscle disease with varying efficacies, depending on the age at which myostatin
inhibition occurs. Here, we describe a one-time gene administration of myostatin-inhibitor-
proteins to enhance muscle mass and strength in normal and dystrophic mouse models for> …
Increasing the size and strength of muscles represents a promising therapeutic strategy for musculoskeletal disorders, and interest has focused on myostatin, a negative regulator of muscle growth. Various myostatin inhibitor approaches have been identified and tested in models of muscle disease with varying efficacies, depending on the age at which myostatin inhibition occurs. Here, we describe a one-time gene administration of myostatin-inhibitor-proteins to enhance muscle mass and strength in normal and dystrophic mouse models for >2 years, even when delivered in aged animals. These results demonstrate a promising therapeutic strategy that warrants consideration for clinical trials in human muscle diseases.
National Acad Sciences