Critical evaluation of the use of cell cultures for inclusion in clinical trials of patients affected by collagen VI myopathies.

doi:10.1002/jcp.23039

Article

Critical evaluation of the use of cell cultures for inclusion in clinical trials of patients affected by collagen VI myopathies.

Journal of cellular physiology - 1 Jul 2012

Sabatelli P, Palma E, Angelin A, Squarzoni S, Urciuolo A, Pellegrini C, Tiepolo T, Bonaldo P, Gualandi F, Merlini L, Bernardi P, Maraldi N M

Abstract excerpt

Collagen VI myopathies (Ullrich congenital muscular dystrophy (UCMD), Bethlem myopathy (BM), and myosclerosis myopathy) share a common pathogenesis, that is, mitochondrial dysfunction due to deregulation of the permeability transition pore (PTP). This effect was first identified in the Col6a1(-/-) mouse model and then in muscle cell cultures from UCMD and BM patients; the normalizing effect of cyclosporin A (CsA)...

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Critical evaluation of the use of cell cultures for inclusion in clinical trials of patients affected by collagen VI myopathies.

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