Splice-region antisense composition and method

   
   

Antisense compositions targeted against an mRNA sequence coding for a selected protein, at a region having its 5' end from 1 to about 25 base pairs downstream of a normal splice acceptor junction in the preprocessed mRNA, are disclosed. The antisense compound is RNase-inactive, and is preferably a phosphorodiamidate-linked morpholino oligonucleotide. Such targeting is effective to inhibit natural mRNA splice processing, produce splice variant mRNAs, and inhibit normal expression of the protein.

Las composiciones de Antisense apuntadas contra un mRNA ordenan la codificación para una proteína seleccionada, en una región que tiene su extremo 5' a partir de la 1 a cerca de 25 pares bajos enes sentido descendiente de una ensambladura normal del aceptador del empalme en el mRNA preprocesado, se divulgan. El compuesto del antisense es RNase-inactivo, y es preferiblemente un oligonucleotide phosphorodiamidate-ligado del morpholino. El tal apuntar es eficaz inhibir el empalme natural del mRNA que procesa, producir mRNAs variables del empalme, e inhibir la expresión normal de la proteína.

 
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