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- W2079077181 abstract "We are now at a point in time where gene therapy is becoming a reality. However, in order to validate these strategies it is essential to have in vitro human cell culture models. The use of patient myoblasts is not always possible due to their drastically decreased proliferative capacity induced by the repeated cycles of degeneration and regeneration. Therefore it is necessary to envisage new in vitro models. In the pioneering studies of Weintraub et al. it had been shown that the forced expression of the myogenic transcription factor myoD was able to convert fibroblasts into myoblasts. In the present study we have developed a universal in vitro model from skin fibroblasts which have been immoralised using hTERT and then converted into myoblasts by a lentivirus containing an inducible myoD contruct. We have then used this model to validate a strategy for exon skipping using fibroblasts isolated from a DMD patient. These fibroblasts were immortalised and then transduced using an inducible myoD construct. We first confirmed the expression of myoD in vitro and the potential of these cells to form differentiated myotubes. These cells were then transfected with an U7 construct to promote exon skipping in the patient. In order to test if these cells could reconstitue muscle fibres in vivo expressing human dystrophin they were injected into cryodamaged TA muscles of immunodeficient RAG−/− gammaC−/− C5−/− mice. Muscle were analysed after 27 days of regeneration and fibres expressing human dystrophin were observed. Therefore this cellular model provides us with an ideal model system to test different therapeutic strategies for various neuromuscular diseases when patient myoblasts are not available." @default.
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- W2079077181 date "2007-10-01" @default.
- W2079077181 modified "2023-09-26" @default.
- W2079077181 title "G.P.3.01 The use of immortalised human fibroblasts from a DMD patient to test exon skipping in vivo" @default.
- W2079077181 doi "https://doi.org/10.1016/j.nmd.2007.06.076" @default.
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