Means and method for inducing exon-skipping

C - Chemistry – Metallurgy – 12 – N

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Details

C12N 15/11 (2006.01) A61K 31/7088 (2006.01) A61K 31/7115 (2006.01) A61K 48/00 (2006.01)

Patent

CA 2652408

In the present invention means and method are provided for optimising exon- skipping using exon-internal AON. We show that skipping efficiencies are improved by targeting putative splicing regulatory sequences (ESEs) within an exon. Such double targeting may be particularly useful for exons with which efficient skipping was difficult to obtain prior to the invention.

Dans la présente invention, des moyens et procédés sont proposés pour optimiser le saut d'exon en utilisant un AON interne à l'exon. Nous montrons que les efficacités de saut sont améliorées en ciblant les séquences régulatrices d'épissage putatives (ESE) dans un exon. Ce double ciblage peut être particulièrement utile pour les exons avec lesquels un saut efficace s'est avéré difficile à obtenir avant l'invention.

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