Gene Therapy - Successess?

[Jon Moulton]

Systemic exon-skipping clinical trial reported for Duchenne muscular dystrophy using Morpholino antisense oligos:
Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study. Cirak S, Arechavala-Gomeza V, Guglieri M, Feng L, Torelli S, Anthony K, Abbs S, Garralda ME, Bourke J, Wells DJ, Dickson G, Wood MJ, Wilton SD, Straub V, Kole R, Shrewsbury SB, Sewry C, Morgan JE, Bushby K, Muntoni F. Lancet. 2011 Jul 22. [Epub ahead of print]
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[Jon Moulton]

The Status of Exon Skipping as a Therapeutic Approach to Duchenne Muscular Dystrophy.
Qi-Long Lu, Toshifumi Yokota, Shin'ichi Takeda, Luis Garcia, Francesco Muntoni, Terence Partridge.
Mol Ther. 2011 January 4; 19(1): 9–15. Published online 2010 October 26. doi: 10.1038/mt.2010.219
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[Jon Moulton]

Looking good so far for Morpholino therapeutics targeting Duchenne muscular dystrophy. Here's a video TV news spot on one family's clinical trial story:
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[detect]

when will gene therapy become profession?

[Stevecolacurcio]

Great ! The gene must be delivered inside the target cells and work properly without causing adverse effects. Thank for sharing.