Methods and compositions for use in spliceosome mediated rna...

C - Chemistry – Metallurgy – 07 – H

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C07H 21/04 (2006.01) A61K 31/711 (2006.01) C12N 15/11 (2006.01) C12N 15/63 (2006.01) C12P 19/34 (2006.01) A61K 48/00 (2006.01)

Patent

CA 2475802

The present invention provides methods and compositions for delivery of synthetic pre-trans-splicing molecules (synthetic PTMs) into a target cell. The compositions of the invention include synthetic pre-trans-splicing molecules (PTMs) with enhanced stability against chemical and enzymatic degradation. The synthetic PTMs are designed to interact with a natural target precursor messenger RNA molecule (target pre-mRNA) and mediate a trans- splicing reaction resulting in the generation of a novel chimeric RNA molecule (chimeric RNA).

La présente invention concerne des méthodes et des compositions servant à apporter des molécules synthétiques de pré-trans-épissage (MPT synthétiques) dans une cellule cible. Les compositions selon l'invention comprennent des molécules synthétiques de pré-trans-épissage présentant une stabilité améliorée contre la dégradation chimique et enzymatique. Les MPT synthétiques sont prévues pour interagir avec une molécule d'ARN messager de précurseur cible naturel (pré-ARNm cible) et induire une réaction de trans-épissage ayant pour résultat de produire une nouvelle molécule d'ARN chimère (ARN chimère).

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