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AVI-4658

 
National Institutes of Health

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eteplirsen

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… Expand
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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
Summary Background We report clinical safety and biochemical efficacy from a dose-ranging study of intravenously administered AVI… Expand
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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… Expand
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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… Expand
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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… Expand
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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…
the dystrophin gene. We are developing methods to deliver therapeutic genes to muscles throughout the body to either replace the… Expand
2010
2010
P01 Current progress with the systemic administration trial of AVI-4658, a novel Phosphorodiamidate Morpholino Oligomer (PMO) skipping dystrophin exon 51 in Duchenne muscular dystrophy (DMD)
 
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
Summary Background Mutations that disrupt the open reading frame and prevent full translation of DMD, the gene that encodes… Expand
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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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