Splice-region antisense composition and method
AssigneeSarepta Therapeutics, Inc.
InventorsPatrick L. Iversen, Robert Hudziak
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Abstract
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.
Claims16
An oligomer of 8 to 40 morpholino subunits connected by phosphorous-containing intersubunit linkages, wherein the 5′ terminal morpholino subunit comprises a group of the formula:
- wherein each R1 is independently a C1-C6 alkyl.
A compound of formula:
- or a pharmaceutically acceptable salt thereof, wherein X is an integer from 3 to 19, and each P is independently a purine or pyrimidine base-pairing moiety selected from adenine, cytosine, guanine, uracil, and thymine.
A compound of formula:
- or a pharmaceutically acceptable salt thereof, wherein X is an integer from 6 to 38, and each P is independently a purine or pyrimidine base-pairing moiety selected from adenine, cytosine, guanine, uracil, and thymine.
Dependencies are inferred from the claim text.