Functional evaluation of gene therapy in a mouse model of Duchenne muscular dystrophy
(1998)
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- Authors
- Supervisors
- Gillis, Jean-Marie
- Abstract
- Although there are many hurdles that must be overcome on the way to developing better gene transfer or utrophin upregulation protocols, it is encouraging to note de speed with which progress is made. Two different gene therapy strategies are now being followed, one based on dystrophin or utrophin gene transfer with the use of viral or not viral vectors, the second based on potential gene upregulation of utrophin. Advances in viral and non viral vector techniques and our improved knowledge of the utrophin promoter provide hope for future treatment of DMD. For dystrophin and utrophin gene transfer, a better understanding of basic muscle cell biology ad issues concerning stability of transgene expression, immune responses, and wide vector distribution to muscle tissue will need careful examination. The second strategy will require the discovery of factors, natural or synthetic, able to switch on strongly and permanently the utrophin gene. It must also be kept in mind that all in vivo work published to date has been almost entirely limited to the mouse model of DMD, and that it will be essential to repeat these studies in larger animal models before human clinical trials can be seriously considered. But scientific and financial investments are really worthwhile, since we demonstrated that these therapies are efficient to obtain a very important functional correction
- Affiliations
Citations
Deconinck, N. (1998). Functional evaluation of gene therapy in a mouse model of Duchenne muscular dystrophy. https://hdl.handle.net/2078.5/111036