Amniotic Fluid Stem Cells Restore the Muscle Cell Niche in a HSA-Cre, SmnF7/F7 Mouse Model§


  • Author contributions: M.P., M.P., and P.D.C: conception and design, data analysis and interpretation, and manuscript writing; C.F.: conception and design, animals genotyping, and data analysis and interpretation; E.B.: collection of amniotic fluid samples; L.U.: collection of muscle samples and data analysis and interpretation; B.B.: muscle mechanics analyses; A.R.: collection of muscle samples; E.T.: animals genotyping; A.C.: image processing; G.F.Z.: financial support; I.A.-S.: data analysis and interpretation; A.R.: in vivo imaging; J.M.: animal model provider; M.C.-C.: data analysis and interpretation and manuscript writing. M.P. and P.D.C. contributed equally to this article.

  • Disclosure of potential conflicts of interest is found at the end of this article.

  • §

    First published online in STEM CELLSEXPRESS May 29, 2012.


Mutations in the survival of motor neuron gene (SMN1) are responsible for spinal muscular atrophy, a fatal neuromuscular disorder. Mice carrying a homozygous deletion of Smn exon 7 directed to skeletal muscle (HSA-Cre, SmnF7/F7 mice) present clinical features of human muscular dystrophies for which new therapeutic approaches are highly warranted. Herein we demonstrate that tail vein transplantation of mouse amniotic fluid stem (AFS) cells enhances the muscle strength and improves the survival rate of the affected animals. Second, after cardiotoxin injury of the Tibialis Anterior, only AFS-transplanted mice efficiently regenerate. Most importantly, secondary transplants of satellite cells (SCs) derived from treated mice show that AFS cells integrate into the muscle stem cell compartment and have long-term muscle regeneration capacity indistinguishable from that of wild-type-derived SC. This is the first study demonstrating the functional and stable integration of AFS cells into the skeletal muscle, highlighting their value as cell source for the treatment of muscular dystrophies. STEM Cells2012;30:1675–1684