Therapeutic molecules generated by trans-splicing

Details Therapeutic molecules generated by trans-splicing Therapeutic molecules generated by trans-splicing

C - Chemistry, Metallurgy

12

N

C12N 15/85 (2006.01) A01N 43/04 (2006.01) A61K 31/70 (2006.01) A61K 31/7088 (2006.01) A61K 31/7105 (2006.01) A61K 31/711 (2006.01) A61K 48/00 (2006.01) A61P 31/12 (2006.01) A61P 35/00 (2006.01) A61P 37/06 (2006.01) A61P 43/00 (2006.01) C07H 21/02 (2006.01) C07H 21/04 (2006.01) C12N 5/10 (2006.01) C12N 15/00 (2006.01) C12N 15/09 (2006.01) C12N 15/10 (2006.01) C12N 15/11 (2006.01) C12N 15/31 (2006.01) C12N 15/63 (2006.01) C12N 15/70 (2006.01) C12N 15/74 (2006.01) C12P 19/34 (2006.01)

Patent

CA 2240494

The molecules and methods of the present invention provide a means for in vivo production of a therapeutic molecule in a selected subset of cells. The pre-therapeutic molecules of the invention are substrates for a trans-splicing reaction between the pre-therapeutic molecules and a pre-mRNA which is uniquely expressed in the specific target cells. The in vivo trans-splicing reaction provides an active therapeutic RNA which is functional as RNA or encodes a protein to be expressed in the target cells. The expression product of the mRNA is a protein of therapeutic value to the cell or a toxin which causes killing of the specific cells.

Ces molécules, et des procédés, permettent de produire in vivo une molécule thérapeutique dans un sous-ensemble choisi de cellules. Ces molécules pré-thérapeutiques décrites constituent des substrats de réaction de trans-épissure avec un pré-ARN messager qui s'exprime exclusivement dans des cellules cibles spécifiques. Cette réaction de trans-épissure in vivo donne un ARN thérapeutique actif qui intervient en tant qu'ARN ou code une protéine devant s'exprimer dans les cellules cibles. Le produit d'expression de l'ARN messager est une protéine présentant un intérêt thérapeutique pour la cellule ou une toxine chargée de tuer des cellules déterminées.

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