Oligonucleotides and methods for the treatment of age-related macular degeneration
AssigneesThe Genera Hospital Corporation, The Schepens Eye Research Institute, Inc.
InventorsAnders M. NAAR, Patricia D'AMORE
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Abstract
Disclosed are oligonucleotides, compositions, and methods that may be useful in the treatment of age-related macular degeneration (AMD). The treatment of age-regulated macular degeneration (AMD) may involve inhibiting an miR-33 target nucleic acid. For example, inhibition of an miR-33 target nucleic acid may be achieved using antisense oligonucleotides targeting an miR-33 target nucleic acid, interfering oligonucleotides targeting an miR-33 target nucleic acid, or recombinant AAV particles including a vector encoding an antisense oligonucleotide or interfering oligonucleotide targeting an miR-33 target nucleic acid.
Claims51
An oligonucleotide comprising a total of 7 to 50 interlinked nucleotides and having a nucleobase sequence comprising at least one bridged nucleic acid and at least 6 contiguous nucleobases complementary to an equal-length portion within an miR-33 target nucleic acid.
A recombinant adeno-associated viral (rAAV) particle comprising a nucleic acid vector that comprises a heterologous nucleic acid region comprising a sequence that encodes an interfering RNA comprising a region complementary to an miR-33 target nucleic acid.
A method of treating age-related macular degeneration in a subject in need thereof, the method comprising administering to the subject a therapeutically effective amount of an miR-33 inhibitor.
A method of treating age-related macular degeneration in a subject in need thereof, the method comprising administering to the subject a therapeutically effective amount of:
- (i) an oligonucleotide comprising a total of 7 to 50 interlinked nucleotides and having a nucleobase sequence comprising at least 6 contiguous nucleobases complementary to an equal-length portion within an miR-33 target nucleic acid;
- or
- (ii) a recombinant adeno-associated viral (rAAV) particles comprising a nucleic acid vector that comprises a heterologous nucleic acid region comprising a sequence that encodes the oligonucleotide.
Dependencies are inferred from the claim text.