Dystrophin expression in host muscle following transplantation of muscle precursor cells modified with the phiC31 integrase
نویسندگان
چکیده
منابع مشابه
Mechanical function of dystrophin in muscle cells
We have directly measured the contribution of dystrophin to the cortical stiffness of living muscle cells and have demonstrated that lack of dystrophin causes a substantial reduction in stiffness. The inferred molecular structure of dystrophin, its preferential localization underlying the cell surface, and the apparent fragility of muscle cells which lack this protein suggest that dystrophin st...
متن کاملEfficient reversal of phiC31 integrase recombination in mammalian cells.
Over the past decade, the integrase enzyme from phage phiC31 has proven to be a useful genome engineering tool in a wide variety of species, including mammalian cells. The enzyme efficiently mediates recombination between two distinct sequences, attP and attB, producing recombinant product sites, attL and attR. The reaction proceeds exclusively in a unidirectional manner, because integrase is u...
متن کاملMini-dystrophin Expression Down-regulates IP3-mediated Calcium Release Events in Resting Dystrophin-deficient Muscle Cells
We present here evidence for the enhancement, at rest, of an inositol 1,4,5-trisphosphate (IP3)-mediated calcium signaling pathway in myotubes from dystrophin-deficient cell lines (SolC1(-)) as compared to a cell line from the same origin but transfected with mini-dystrophin (SolD(+)). With confocal microscopy, the number of sites discharging calcium (release site density [RSD]) was quantified ...
متن کاملEnhancement of plasmid-mediated gene therapy for muscular dystrophy by directed plasmid integration.
Plasmid-mediated gene therapy can restore dystrophin expression in skeletal muscle in the mdx mouse, a model of Duchenne muscular dystrophy. However, sufficient long-term expression and distribution of dystrophin remain a hurdle for translating this technology into a viable treatment for Duchenne muscular dystrophy. To improve plasmid-mediated gene therapy for muscle diseases, we studied the ef...
متن کاملThe promotion of a functional fibrosis in skeletal muscle with volumetric muscle loss injury following the transplantation of muscle-ECM.
Tissue engineering strategies that primarily use biological extracellular matrices (ECMs) with or without the inclusion of a stem or progenitor cell source are under development for the treatment of trauma resulting in the loss of a large volume of skeletal muscle (i.e., volumetric muscle loss; VML). The explicit goal is to restore functional capacity to the injured tissue by promoting generati...
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ژورنال
عنوان ژورنال: Gene Therapy
سال: 2006
ISSN: 0969-7128,1476-5462
DOI: 10.1038/sj.gt.3302887