Study with Funding from FSMA Shows a Muscle Specific Intervention Improves Phenotype in a Mouse Model of SMA
Press Release | February 18, 2011
Dr. Charlotte Sumner and colleagues at Johns Hopkins University publish a study in the journal Human Molecular Genetics showing improvement in survival but not motor function in a severe model of Spinal Muscular Atrophy by increasing the levels of the muscle modulator Insulin-like growth factor 1 (IGF-1).
Spinal muscular atrophy (SMA) is an inherited motor neuron disease caused by the mutation of the survival motor neuron 1 (SMN1) gene and deficiency of the SMN protein. Severe SMA mice have abnormal motor function and small, immature myofibers early in development suggesting that SMN protein deficiency results in retarded muscle growth. Insulin-like growth factor 1 (IGF-1) stimulates myoblast proliferation, induces myogenic differentiation, and generates myocyte hypertrophy in vitro and in vivo.
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