Journal
BIOMATERIALS
Volume 75, Issue -, Pages 313-326Publisher
ELSEVIER SCI LTD
DOI: 10.1016/j.biomaterials.2015.10.029
Keywords
Duchenne muscular dystrophy; Sertoli cells; SPF; Microencapsulation; Engraftment; Heregulin
Funding
- Regione Umbria, Italy (POR UMBRIA FSE)
- AFM (Association Francaise contre les Myopathies) fellowship
- AFM [15679]
- Ministero dell'Universita e della Ricerca [PRIN 2010R8JK2X_004, PRIN 2009WBFZYM_002]
- Fondazione Cassa di Risparmio di Perugia [2009.020.0021, 2012.0241.021]
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Duchenne muscular dystrophy (DMD) is a genetic disease characterized by progressive muscle degeneration leading to impaired locomotion, respiratory failure and premature death. In DMD patients, inflammatory events secondary to dystrophin mutation play a major role in the progression of the pathology. Sertoli cells (SeC) have been largely used to protect xenogeneic engraftments or induce trophic effects thanks to their ability to secrete trophic, antiinflammatory, and immunomodulatory factors. Here we have purified SeC from specific pathogen-free (SPF)-certified neonatal pigs, and embedded them into clinical grade alginate microcapsules. We show that a single intraperitoneal injection of micro-encapsulated SPF SeC (SeC-MC) in an experimental model of DMD can rescue muscle morphology and performance in the absence of pharmacologic immunosuppressive treatments. Once i.p. injected, SeC-MC act as a drug delivery system that modulates the inflammatory response in muscle tissue, and upregulates the expression of the dystrophin paralogue, utrophin in muscles through systemic release of heregulin-beta 1, thus promoting sarcolemma stability. Analyses performed five months after single injection show high biocompatibility and long-term efficacy of SeC-MC. Our results might open new avenues for the treatment of patients with DMD and related diseases. (C) 2015 Elsevier Ltd. All rights reserved.
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