Another alternative strategy to gene therapy is exon skipping which differs from gene therapy in that it focuses on the gene transcript rather than the gene itself (McClorey et al., 2005). Exon skipping prevents the transcription of the exon containing the mutation. Exon skipping is a process by which synthetic DNA molecules, antisense, are utilized to create a bridge by which the dysfunctional parts of the gene can be skipped over (Partridge, 2010). This intervention has been found to be particularly useful when treating Duchenne MD in laboratory animals and experts are hoping that this will correlate to human subjects as well.

Cell therapy has also shown promise in the treatment of MD. In this approach cells can be taken either from a donor that has healthy formations of the mutated gene or by utilizing repaired cells of the individual with MD (Cossu and Sampaolesi, 2004). The use of the...
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