Transplacental delivery of oligonucleotides
AssigneeQIK Technologies, Inc.
InventorsPeter C. Roberts, Samuel E. Driver
Life of this patent
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Abstract
This invention provides synthetic oligonucleotides that are useful for transplacental delivery, the oligonucleotides being DNA or RNA or both, preferably between 12-35 nucleotides in length, having a stabilized and charged backbone and at least one chemically modified base or sugar moiety, wherein the modified base or sugar moiety facilitates transplacental delivery.
Claims10
A method of determining the function of a gene expressed in a mouse, comprising:
- (a) transplacentally delivering to a mouse embryo a synthetic oligonucleotide effective in modulating the expression of the gene by systemically administering to a pregnant mouse in which the embryo is present the oligonucleotide in a pharmaceutically acceptable carrier, wherein the synthetic oligonucleotide comprises:
- (i) RNA or a combination of RNA and DNA;
- (ii) a stabilized backbone, comprising phophorothioate internucleotide linkages;
- and
- (iii) at least one 2′-O-alkyl ribonucleotide, the alkyl being a saturated alkyl;
- and
- (b) determining whether the phenotype of the embryo is altered, the altered phenotype of the embryo being indicative of the function of the gene.
A method of delivering a synthetic oligonucleotide to a mouse embryo, comprising systemically administering to a pregnant mouse in which the embryo is present the oligonucleotide and a pharmaceutically acceptable carrier, wherein the synthetic oligonucleotide comprises:
- (a) RNA or a combination of RNA and DNA;
- (b) a stabilized backbone, comprising phophorothioate internucleotide linkages;
- and
- (c) at least one 2′-O-alkyl ribonucleotide, the alkyl being a saturated alkyl;
- wherein the oligonucleotide passes through the placenta into the embryo.
Dependencies are inferred from the claim text.