Amniotic fluid stem cells restore the muscle cell niche in a HSA-Cre, SmnF7/F7 mouse model

Martina Piccoli, Chiara Franzin, Enrica Bertin, Luca Urbani, Bert Blaauw, Andrea Repele, Elisa Taschin, Angelo Cenedese, Giovanni Franco Zanon, Isabelle André-Schmutz, Antonio Rosato, Judith Melki, Marina Cavazzana-Calvo, Michela Pozzobon, Paolo De Coppi

Research output: Contribution to journalArticlepeer-review


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, Smn F7/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.

Original languageEnglish
Pages (from-to)1675-1684
Number of pages10
JournalStem Cells
Issue number8
Publication statusPublished - Aug 2012


  • Amniotic fluid stem cells
  • Muscle dystrophy
  • Muscle stem cell niche
  • Spinal muscular atrophy

ASJC Scopus subject areas

  • Cell Biology
  • Developmental Biology
  • Molecular Medicine


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