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AVI-4658

National Institutes of Health

Papers overview

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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… 
2011
2011
AVI-4658 is a phosphorodiamidate morpholino oligomer (PMO) drug designed to restore dystrophin expression in a subset of patients… 
2011
2011
AVI-4658 is a phosphorodiamidate morpholino oligomer (PMO) designed to induce skipping of dystrophin exon 51 and restore its… 
2010
2010
Duchenne muscular dystrophy (DMD) is caused by dystrophin gene mutations. Restoration of dystrophin by exon skipping was… 
Review
2009
Review
2009
Purpose of reviewDuchenne muscular dystrophy is a progressive muscle degenerative disease caused by dystrophin mutations. The…