Enhanced expression of alternative spliced uromodulin for therapeuticuse
AssigneeThe Trustees of Indiana University
InventorsTarek Maurice Ashkar, Azuma Nanamatsu
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Abstract
A method of enhancing the presence of alternatively-spliced UMOD protein by inducing exon-skipping of a AS-UMOD m-RNA to protect TAL cells of kidneys in a patient suffering from acute kidney injury or chronic kidney disease.
Claims17
A method of inducing exon-skipping of a AS-UMOD m-RNA in a TAL cell, the method comprising delivering to the cell a splice-switching antisense oligonucleotides (SSO) comprising a sequence selected from the group consisting of SEQ ID NOs:
- 14, 15, and 16, wherein the antisense oligonucleotide is up to 30 nucleotides in length and is capable of inducing exon-skipping of the UMOD pre-mRNA.
A method for enhancing endogenous alternatively spliced uromodulin (AS-UMOD) expression in a subject, comprising
- administering splice-switching antisense oligonucleotides (SSO) comprising a sequence selected from the group consisting of SEQ ID NOs:
- 14, 15, and 16 to a subject.
A spice-switching antisense oligonucleotide (SSO) consisting of a sequence that has at least 90% identical over the entire length of SEQ ID NO:
- 14, 15 or 16.
A pharmaceutical composition comprising a pharmaceutically acceptable carrier and two or more antisense oligonucleotides complementary to AS-UMOD, wherein at least one of said antisense oligonucleotides thereof comprises a nucleic acid sequence as set forth in any one of SEQ ID Nos:
- 14, 15 or 16.
Dependencies are inferred from the claim text.