Gene therapy clinical trials for muscular dystrophies

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Abstract

The major challenge for therapy of Duchenne and related muscular dystrophies is the need to treat multiple muscles for clinical benefit. After many years of laboratory studies developing systems for gene transfer into muscle, the last 5 years have seen major steps forward with the generation of genetic treatments that can be administered systemically. Consequently, a number of different approaches have been taken through to clinical trials, including adeno-associated viral vector mediated gene delivery, plasmid mediated gene delivery, and the use of antisense oligonucleotides to modify splicing of the primary transcript to restore the reading frame. Cell transfer can also be used as a means of gene delivery. Associated with clinical trials has been the consideration of appropriate pre-clinical safety testing and outcome measures for assessing efficacy for regulatory approval. Although many of the trials look promising and offer hope for the future, it is important to understand that therapeutic approval can take considerable time and requires significant funding and input from biotechnology/pharmaceutical companies.

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APA

Wells, D. J. (2010). Gene therapy clinical trials for muscular dystrophies. In Muscle Gene Therapy (pp. 257–267). Springer New York. https://doi.org/10.1007/978-1-4419-1207-7_16

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