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Gene Therapy Discuss gene therapy issues, methods and protocols for viral gene therapy, problems and solutions.


Gene Therapy - Successess?

Gene Therapy - Successess? - Gene Therapy

Gene Therapy - Successess? - Discuss gene therapy issues, methods and protocols for viral gene therapy, problems and solutions.


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  #21  
Old 07-26-2011, 07:04 PM
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Default Re: Gene Therapy - Successess?

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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  #22  
Old 01-09-2012, 07:24 PM
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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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  #23  
Old 08-15-2012, 04:05 PM
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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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  #24  
Old 10-06-2012, 02:43 PM
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Default Re: Gene Therapy - Successess?

when will gene therapy become profession?
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  #25  
Old 01-03-2013, 11:03 AM
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Default Re: Gene Therapy - Successess?

Great ! The gene must be delivered inside the target cells and work properly without causing adverse effects. Thank for sharing.
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  #26  
Old 08-01-2013, 06:34 PM
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Post Re: Gene Therapy - Successess?

Mendell J, Rodino-Klapac LR, Sahenk Z, Roush K, Bird L, Lowes LP, Alfano L, Gomez AM, Lewis S, Kota J, Malik V, Shontz K, Walker CM, Flanigan KM, Kean JR, Allen HD, Shilling C, Melia KR, Sazani P, Saoud JB, Kaye EM, The Eteplirsen Study Group. [Only registered users see links. ]. Ann Neurol. 2013;[Epub ahead of print] doi:10.1002/ana.23982
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