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Last Updated: March 28, 2024

Details for Patent: 6,784,291


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Title: Splice-region antisense composition and method
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.
Inventor(s): Iversen; Patrick L. (Corvallis, OR), Hudziak; Robert (Blodgett, OR)
Assignee: AVI BioPharma, Inc. (Corvallis, OR)
Filing Date:May 04, 2001
Application Number:09/848,868
Claims:1. An antisense compound having an uncharged morpholino backbone and the base sequence presented as SEQ ID NO: 35.

2. The compound of claim 1, wherein said backbone comprises morpholino subunits, as shown in the structure below, ##STR1##

where P.sub.i is a purine or pyrimidine base-pairing moiety effective to bind to a base in a polynucleotide, and uncharged phosphorus-containing linkages, one to three atoms long, joining the morpholino nitrogen of one subunit to the 5' exocyclic carbon of an adjacent subunit.

3. The compound of claim 2, wherein the each said phosphorus-containing linkage is a phosphorodiamidate linkage as represented by --P(=Z)(X)--Y--, where X=NH.sub.2, NHR, or NRR', Y=O, and Z=O, or where X=l OR, Y=NH or NR', and Z=O, and R and R' are groups which do not interfere with target binding.

4. The compound of claim 3, wherein R and R' are moieties independently selected from alkyl, polyalkyleneoxy, and a combination thereof, which may be substituted with one or more groups selected from hydroxy, alkoxy, amino, alkylamino, thiol, alkanethiol, halogen, oxo, carboxylic acid, carboxylic ester, and inorganic ester.

5. The compound of claim 4, wherein each said moiety R and R', independent of substitution, is from 1 to 6 atoms long.

6. The compound of claim 3, wherein NRR' represents a nitrogen heterocycle having 5-7 ring atoms selected from nitrogen, carbon, oxygen, and sulfur, and having at least as many carbon ring atoms as non-carbon ring atoms.

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