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Article

Activin Receptor Type IIB Inhibition Improves Muscle Phenotype and Function in a Mouse Model of Spinal Muscular Atrophy.

PloS one - 1 Jan 2016

Liu Min, Hammers David W, Barton Elisabeth R, Sweeney H Lee

Abstract excerpt

Spinal muscular atrophy (SMA) is a devastating neurodegenerative disorder that causes progressive muscle atrophy and weakness. Using adeno-associated virus-mediated gene transfer, we evaluated the potential to improve skeletal muscle weakness via systemic, postnatal inhibition of either myostatin or all signaling via the activin receptor type IIB (ActRIIB). After demonstrating elevated p-SMAD3 content and...

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Identifiers

PMID: 27870893
DOI: 10.1371/journal.pone.0166803
PMCID: PMC5117715

Genes

SMAD3

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