Sglt-2 targeting oligonucleotides
AssigneeUniversity of Massachusetts
InventorsAnastasia Khvorova, Ken Yamada, Vignesh Narayan Hariharan, Julianna Buchwald
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Abstract
This disclosure relates to oligonucleotides with internucleotide linkage modifications and used thereof for treating and preventing diseases. Particularly, this disclosure relates to SLC5A2 targeting sequences, and methods for treating and preventing kidney diseases using same.
Claims33
An oligonucleotide comprising a 5′ end, a 3′ end, and a sequence substantially complementary to a solute carrier family 5 member 2 (SLC5A2) nucleic acid sequence of any one of SEQ ID NOs:
- 1-24.
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A method of treating or managing a disease associated with solute carrier family 5 member 2 (SLC5A2), the method comprising administering to a patient in need of such treatment or management a therapeutically effective amount of an oligonucleotides comprising a 5′ end, a 3′ end, and a sequence substantially complementary to a SLC5A2 nucleic acid sequence;
- wherein the oligonucleotide inhibits a SLC5A2 gene expression by at least about 50% for four weeks post administration.
A vector comprising a regulatory sequence operably linked to a nucleotide sequence that encodes an oligonucleotide substantially complementary to a solute carrier family 5 member 2 (SLC5A2) nucleic acid sequence of any one of SEQ ID NOs:
- 1-24, optionally wherein the oligonucleotide inhibits expression of the SLC5A2 nucleic acid sequence by at least 30%, at least 50%, or at least 80%.
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Dependencies are inferred from the claim text.