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AVI-4658

National Institutes of Health

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Papers overview

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2013
2013
Can manipulation of splicing offer gene therapy possibilities to those with tumour-prone disorders?
Can manipulation of splicing offer gene therapy possibilities to those with tumour-prone disorders? 
Highly Cited
2012
Highly Cited
2012
Restoration of the dystrophin-associated glycoprotein complex after exon skipping therapy in Duchenne muscular dystrophy.
We previously conducted a proof of principle; dose escalation study in Duchenne muscular dystrophy (DMD) patients using the… 
Highly Cited
2011
Highly Cited
2011
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
2011
2011
Repeat-Dose Toxicology Evaluation in Cynomolgus Monkeys of AVI-4658, a Phosphorodiamidate Morpholino Oligomer (PMO) Drug for the Treatment of Duchenne Muscular Dystrophy
AVI-4658 is a phosphorodiamidate morpholino oligomer (PMO) drug designed to restore dystrophin expression in a subset of patients… 
2011
2011
Chemical and Mechanistic Toxicology Evaluation of Exon Skipping Phosphorodiamidate Morpholino Oligomers in mdx Mice
AVI-4658 is a phosphorodiamidate morpholino oligomer (PMO) designed to induce skipping of dystrophin exon 51 and restore its… 
2010
2010
Safety Pharmacology and Genotoxicity Evaluation of AVI-4658
Duchenne muscular dystrophy (DMD) is caused by dystrophin gene mutations. Restoration of dystrophin by exon skipping was… 
2010
2010
O.16 Current progress and preliminary results with the systemic administration trial of AVI-4658, a novel phosphorodiamidate morpholino oligomer (PMO) skipping dystrophin exon 51 in Duchenne muscular…
Highly Cited
2009
Highly Cited
2009
Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study
Review
2009
Review
2009
Emerging genetic therapies to treat Duchenne muscular dystrophy
Purpose of reviewDuchenne muscular dystrophy is a progressive muscle degenerative disease caused by dystrophin mutations. The… 
2009
2009
T.O.3 Restoration of dystrophin expression in Duchenne muscular dystrophy: A single blind, placebo-controlled dose escalation study using morpholino oligomer AVI-4658
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