Abstract
The muscular dystrophies collectively represent a major health challenge, as few significant treatment options currently exist for any of these disorders. Recent years have witnessed a proliferation of novel approaches to therapy, spanning increased testing of existing and new pharmaceuticals, DNA delivery (both anti-sense oligonucleotides and plasmid DNA), gene therapies and stem cell technologies. While none of these has reached the point of being used in clinical practice, all show promise for being able to impact different types of muscular dystrophies. Our group has focused on developing direct gene replacement strategies to treat recessively inherited forms of muscular dystrophy, particularly Duchenne and Becker muscular dystrophy (DMD/BMD). Both forms of dystrophy are caused by mutations in the dystrophin gene and all cases can in theory be treated by gene replacement using synthetic forms of the dystrophin gene. The major challenges for success of this approach are the development of a suitable gene delivery shuttle, generating a suitable gene expression cassette able to be carried by such a shuttle, and achieving safe and effective delivery without elicitation of a destructive immune response. This review summarizes the current state of the art in terms of using adeno-associated viral vectors to deliver synthetic dystrophin genes for the purpose of developing gene therapy for DMD.
Keywords: Adeno-associated viral vector, duchenne muscular dystrophy, dystrophin, gene therapy, immune response, micro-dystrophin, micro-utrophin, DMD, AAV, GRMD
Current Gene Therapy
Title:Gene Replacement Therapies for Duchenne Muscular Dystrophy Using Adeno-Associated Viral Vectors
Volume: 12 Issue: 3
Author(s): Jane T. Seto, Julian N. Ramos, Lindsey Muir, Jeffrey S. Chamberlain and Guy L. Odom
Affiliation:
Keywords: Adeno-associated viral vector, duchenne muscular dystrophy, dystrophin, gene therapy, immune response, micro-dystrophin, micro-utrophin, DMD, AAV, GRMD
Abstract: The muscular dystrophies collectively represent a major health challenge, as few significant treatment options currently exist for any of these disorders. Recent years have witnessed a proliferation of novel approaches to therapy, spanning increased testing of existing and new pharmaceuticals, DNA delivery (both anti-sense oligonucleotides and plasmid DNA), gene therapies and stem cell technologies. While none of these has reached the point of being used in clinical practice, all show promise for being able to impact different types of muscular dystrophies. Our group has focused on developing direct gene replacement strategies to treat recessively inherited forms of muscular dystrophy, particularly Duchenne and Becker muscular dystrophy (DMD/BMD). Both forms of dystrophy are caused by mutations in the dystrophin gene and all cases can in theory be treated by gene replacement using synthetic forms of the dystrophin gene. The major challenges for success of this approach are the development of a suitable gene delivery shuttle, generating a suitable gene expression cassette able to be carried by such a shuttle, and achieving safe and effective delivery without elicitation of a destructive immune response. This review summarizes the current state of the art in terms of using adeno-associated viral vectors to deliver synthetic dystrophin genes for the purpose of developing gene therapy for DMD.
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T. Seto Jane, N. Ramos Julian, Muir Lindsey, S. Chamberlain Jeffrey and L. Odom Guy, Gene Replacement Therapies for Duchenne Muscular Dystrophy Using Adeno-Associated Viral Vectors, Current Gene Therapy 2012; 12 (3) . https://dx.doi.org/10.2174/156652312800840603
DOI https://dx.doi.org/10.2174/156652312800840603 |
Print ISSN 1566-5232 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5631 |
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