Ocular delivery of oligonucleotides
AssigneeUniversity of Massachusetts
InventorsClaudio Punzo, Anastasia Khvorova, Dimas Echeverria Moreno, Annabelle Biscans, Julia F. Alterman, Matthew Hassler, Shun-Yun Cheng
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Abstract
Provided herein are conjugated oligonucleotides that are characterized by efficient and specific eye distribution.
Claims31
A method for delivering an oligonucleotide conjugate to an eye of a subject, the method comprising administering the oligonucleotide conjugate to the subject, wherein the oligonucleotide conjugate comprises:
- i) an oligonucleotide comprising a 5′ and a 3′ end and complementarity to a target nucleic acid;
- and
- ii) a functional moiety linked to the oligonucleotide, wherein the functional moiety comprises any one of triple amine, retinoic acid, docosahexaenoic acid (DHA), docosanoic acid (DCA), α-tocopheryl succinate, or lithocholic acid (LA).
A method for delivering a branched oligonucleotide to an eye of a subject, the method comprising administering the branched oligonucleotide to the subject, wherein the branched oligonucleotide comprises two or more oligonucleotides, each oligonucleotide comprising a 5′ and a 3′ end and complementarity to a target nucleic acid.
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An oligonucleotide conjugate comprising:
- i) an oligonucleotide comprising a 5′ end, a 3′ end, and complementarity to a target nucleic acid;
- and
- ii) a di-docosahexaenoic acid (di-DHA) functional moiety linked to the oligonucleotide.
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An oligonucleotide conjugate comprising:
- i) an oligonucleotide comprising a 5′ end, a 3′ end, and complementarity to a target nucleic acid;
- and
- ii) a triple amine functional moiety linked to the oligonucleotide.
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Dependencies are inferred from the claim text.