Journal
EXPERT OPINION ON BIOLOGICAL THERAPY
Volume 8, Issue 8, Pages 1051-1061Publisher
TAYLOR & FRANCIS LTD
DOI: 10.1517/14712598.8.8.1051
Keywords
adult stem cells; AON; DMD; exon skipping; muscular dystrophies; viral vectors
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Background: Muscular dystrophies are characterized by primary wasting of skeletal muscle. Mutations in the dystrophin gene cause hereditary muscular diseases such as Becker muscular dystrophy (BMD) and Duchenne muscular dystrophy (DMD), the most severe form. Characterization of the dystrophin gene and evidence that different types of adult stem cells are capable of muscle regeneration has lead to the development of potential gene therapy and stem cell treatments for DMD. Objectives: The main goal is to combine gene modification strategies with cell-mediated therapies. This approach could permit autologous transplantation of cells, minimizing the risk of implant rejection. Results/conclusion: The combination of gene and stem cell approaches seems to be most promising, particularly intra-arterial injections of the patient's own stem cells transduced by antisense oligonucleotide technology. This approach should offer the chance to distribute the autologous corrected stem cells to the whole body musculature providing a clinical benefit for dystrophic patients.
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