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Restoration of dystrophin expression in cultured hybrid myotubes
Author(s) -
Radojevic V.,
Oppliger C.,
Gaschen F.,
Burgunder J.M.
Publication year - 2002
Publication title -
neuropathology and applied neurobiology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.538
H-Index - 95
eISSN - 1365-2990
pISSN - 0305-1846
DOI - 10.1046/j.1365-2990.2002.00409.x
Subject(s) - dystrophin , utrophin , myogenesis , duchenne muscular dystrophy , myocyte , biology , microbiology and biotechnology , genetics
Absence of dystrophin, as found in Duchenne boys, mdx mice and HFMD cats, leads to destabilization of the sarcolemmal‐associated protein complex. Gene and cell therapy strategies aim to restore the dystrophin‐associated protein complex. In order to better understand the cellular events involved in such therapy in feline and human muscular dystrophy, we asked whether dystrophin‐deficient myoblasts would fuse with myoblasts expressing normal dystrophin, and whether the complex would be restored after such a fusion. Cat and human myoblasts were isolated from skeletal muscle of normal subjects and of patients with dystrophin deficiency and proliferated well. After co‐culture with normal myoblasts, they fused to form hybrid myotubes. These hybrid myotubes expressed dystrophin, utrophin and dystrophin‐ associated proteins. Expression of these proteins were restored also in the vicinity of nuclei from dystrophin‐deficient donors. These results demonstrate that dystrophin can be expressed and handled normally by hybrid myotubes. They show that myoblasts with a normal dystrophin gene can restore dystrophin expression in dystrophin‐deficient myoblasts.