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`MULTEPLE EXON SKiPPiNG COMPOSiTiONS FOR DMD
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`CRQSS—REEERENCE TO RELATED APPLESATiON
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`This appiication ciairns the benefit under 35 USE. § iigte) of US.
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`Provisionai patent Appiication No. 61/198,416 tiied Qctoper 24, 2908; wherein this
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`provisionai appiication is incorporated herein by reference in its entirety.
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`STATEMENT REGARDiNQ SEQUENCE LiSTihiG
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`The Sequence Listing associated with this appiication is provided in
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`text format in iieu of a paper copy, and is hereby incorporated by reference into the
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`specification.
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`The name of the text
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`tiie containing the Sequence Listing is
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`10
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`i2G178m4’iGMSEQUENCEmLiSTENG.txt. The text tits is 15? K8, was created on
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`Qctoper 23, 2098 and is being submitted eiectronicaiiy via EESuWep.
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`FEELD OF THE ENVENTEON
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`The present
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`invention reiates to novei antisense compounds and
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`compositions suitabie for 'iaciiitating exon skipping in the human dystrophin gene.
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`15
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`it aiso provides methods for
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`inducing exon skipping using the antisense
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`compositions adapted for use in the methods or the invention.
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`BACKGROUND OF THE ENVENTEQN
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`Antisense technoiogies are being deveioped using a range of
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`chemistries to affect gene expression at a variety of different ieveis (transcription,
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`spiicing, stahiiity, transiationi. Much of that research has focused on the use of
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`antisense compounds to correct or compensate tor abnormai or disease"
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`associated genes in a wide range of indications. Antisense moiecuies are ahie to
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`inhibit gene expression with specificity, and because of this, many research efforts
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`concerning oiigonucieotides as moduiators of gene expression have focused on
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`inhibiting the expression of targeted genes or the function of cismacting eiements.
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`2t)
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`25
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