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AVI-4658

 
National Institutes of Health

Papers overview

Semantic Scholar uses AI to extract papers important to this topic.
2013
2013
Can manipulation of splicing offer gene therapy possibilities to those with tumour-prone disorders? 
Highly Cited
2012
Highly Cited
2012
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
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
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
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
Duchenne muscular dystrophy (DMD) is caused by dystrophin gene mutations. Restoration of dystrophin by exon skipping was… Expand
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2010
2010
the dystrophin gene. We are developing methods to deliver therapeutic genes to muscles throughout the body to either replace the… Expand
Highly Cited
2009
Highly Cited
2009
Summary Background Mutations that disrupt the open reading frame and prevent full translation of DMD, the gene that encodes… Expand
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