Optimization of U7snRNA Cassettes for Expression of Therapeutic Antisense Sequences

Pre-mRNA maturation can be modulated using antisense oligonudeotides. These approaches have been recently used in clinical trials where exon-skipping is induced on a mutated DMD pre-mRNA in order to reestablish the translational reading frame. Stable delivery of antisense sequences can be obtained w...

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Veröffentlicht in:	Human gene therapy 2010-04, Vol.21 (4), p.524-524
Hauptverfasser:	Eckenfelder, A, Tordo, J, Jollet, A, Garcia, L, Danos, O
Format:	Artikel
Sprache:	eng
Online-Zugang:	Volltext
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