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Mini-Dystrophin Genes Effective in Mouse Model of Duchenne Muscular Dystrophy
In finding a way to treat Duchenne muscular dystrophy (MDM) the major strategy being studied
today is that of gene therapy - i.e. the introduction of a normal gene to replace the abnormal gene in muscle.
The replacement genes have to be carried into the muscle by a vector, and certain viruses have been found to be
effective as vectors.
One of the main problems in the gene therapy of DMD is that the DMD gene (dystrophin gene) is very large, and
hardly any virus can cope with this large size. For this reason, if the DMD gene can be made smaller (i.e. a
mini-dystrophin gene) and yet be effective, it would be easier to transport the gene in to muscle. Another
reason to try to construct such a mini-dystrophin gene is that it can then be carried by a small virus called
the AAV virus. This AAV virus has many advantages, including increased safety and efficiency.
In a December 2000 issue of the Proceedings of the National Academy of Sciences, USA, researchers from the
University of Pittsburgh led by Dr. Xiao Xiao, reported that they have constructed several mini-dystrophin genes
that were readily packaged into the AAV virus. They found two of these mini-dystrophin genes were efficiently
transferred into the muscles of mice with a mouse form of DMD. These mini-dystrophin genes not only remain
stable within the muscle fibres, they also corrected the abnormal structural or pathological changes in the
muscle. The researchers expressed the hope that such mini-dystrophin genes can eventually be studied in patients
with Duchenne or Becker muscular dystrophy.
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