Duchenne muscular dystrophy (DMD) is a recessive X-linked form of muscular dystrophy caused by mutations in the dystrophin gene. The absence of the protein caused fibrotic tissue deposition and adipose infiltration into the muscle until complete replacement of original tissue at later stages of the disease and DMD patients die from heart and respiratory failure. Unravelling the precise cellular origin and molecular mechanism of fibrotic and adipogenic tissue within a degenerating human DMD muscle is crucial to understand if the dystrophic muscle environment could influence the outcome of stem cells based therapy and to improve future treatments. Recently, we isolated through FACS sorting from dissociated muscular biopsies two MSC populations that express or not the CD133 antigen. These populations could be responsible for muscle regeneration exhaustion and adipogenic tissue deposition in DMD. We identified miRNAs involved in in-vitro differentiation process and in DMD muscle degeneration in the isolated CD133+ and CD133- hmMSCs and unravelled gene regulatory networks that miRNAs control in DMD. In a clinical prospective, we also tested the therapeutic value of targeting miRNAs to enhance transduction efficiency of hmMSCs into muscle using a dystrophic animal model (scid-mdx mice).

Identification of miRNAs to improve myogenic differentiation of Mesenchymal Stem Cells as regenerative therapy for Duchenne muscular dystrophy / S. Banfi, P. Razini, S. Erratico, M. Meregalli, M. Belicchi, Y. Torrente. ((Intervento presentato al convegno Myology tenutosi a Lyon nel 2016.

Identification of miRNAs to improve myogenic differentiation of Mesenchymal Stem Cells as regenerative therapy for Duchenne muscular dystrophy

M. Meregalli;M. Belicchi;Y. Torrente
2016

Abstract

Duchenne muscular dystrophy (DMD) is a recessive X-linked form of muscular dystrophy caused by mutations in the dystrophin gene. The absence of the protein caused fibrotic tissue deposition and adipose infiltration into the muscle until complete replacement of original tissue at later stages of the disease and DMD patients die from heart and respiratory failure. Unravelling the precise cellular origin and molecular mechanism of fibrotic and adipogenic tissue within a degenerating human DMD muscle is crucial to understand if the dystrophic muscle environment could influence the outcome of stem cells based therapy and to improve future treatments. Recently, we isolated through FACS sorting from dissociated muscular biopsies two MSC populations that express or not the CD133 antigen. These populations could be responsible for muscle regeneration exhaustion and adipogenic tissue deposition in DMD. We identified miRNAs involved in in-vitro differentiation process and in DMD muscle degeneration in the isolated CD133+ and CD133- hmMSCs and unravelled gene regulatory networks that miRNAs control in DMD. In a clinical prospective, we also tested the therapeutic value of targeting miRNAs to enhance transduction efficiency of hmMSCs into muscle using a dystrophic animal model (scid-mdx mice).
mar-2016
Settore MED/26 - Neurologia
Identification of miRNAs to improve myogenic differentiation of Mesenchymal Stem Cells as regenerative therapy for Duchenne muscular dystrophy / S. Banfi, P. Razini, S. Erratico, M. Meregalli, M. Belicchi, Y. Torrente. ((Intervento presentato al convegno Myology tenutosi a Lyon nel 2016.
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/2434/372323
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