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Long-term Exon Skipping Studies With 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides in Dystrophic Mouse Models

Antisense-mediated exon skipping for Duchenne muscular dystrophy (DMD) is currently tested in phase 3 clinical trials. The aim of this approach is to modulate splicing by skipping a specific exon to reframe disrupted dystrophin transcripts, allowing the synthesis of a partly functional dystrophin pr...

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Bibliografiska uppgifter
Huvudupphovsmän:	Christa L Tanganyika-de Winter (Författare, medförfattare), Hans Heemskerk (Författare, medförfattare), Tatyana G Karnaoukh (Författare, medförfattare), Maaike van Putten (Författare, medförfattare), Sjef J de Kimpe (Författare, medförfattare), Judith van Deutekom (Författare, medförfattare), Annemieke Aartsma-Rus (Författare, medförfattare)
Materialtyp:	Bok
Publicerad:	Elsevier, 2012-01-01T00:00:00Z.
Ämnen:	article
Länkar:	Connect to this object online.
Taggar:	Lägg till en tagg Inga taggar, Lägg till första taggen!

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