making muscle wasting history

Exon Skipping Review April 2009

• Last Edited: 8 May 2009
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This review is an excellent way to start undersatnding exon skipping and also to catch up with the very latest developments. You can also view all of Günter Scheuerbrandt’s other excellent r...

Tags: exon skipping, genes, proteins, gene transfer.

Exon skipping with U7 gene transfer

• Last Edited: 6 Jul 2009
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This is a gene therapy approach for Duchenne muscular dystrophy (DMD), based on the exon-skipping strategy and is currently under development. This approach consists of interacting with the splici...

Tags: gene, DNA, exon skipping, rna, U7, gene transfer.

Transfer of the dystrophin gene, with a viral vector

• Last Edited: 7 Jul 2009
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Adeno-associated virus (AAV) vector is a potential tool of gene therapy for treatment of genetic neuromuscular disorders. It is a non-pathogenic and replication-defective viral vector without any ...

Tags: gene transfer, dystrophin, AAv, viral vector.

ultrasound and microbubble mediated in vivo gene transfer.

• Last Edited: 24 Sep 2010
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Studies have shown that ultrasound, used either alone or in combination with microbubble contrast agents, can increase cell membrane permeability to plasmid DNA. Because ultrasound is a non-painfu...

Tags: gene transfer, gene therapy, dystrophic muscles, ultrasound.