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US 2024/0352456 A1Published application
Compositions and methods for treating sickle cell diseases
AssigneeThe United States of America, as represented by the Secretary, Department of Health and Human Services
InventorsJohn Fitzgerald TISDALE, Bjorg GUDMUNDSDOTTIR, Laxminath TUMBURU
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Abstract
The present invention provides ribonucleotide agents that decrease expression of RIOK3 for the treatment of a sickle cell disease.
Claims29
A ribonucleotide agent that decreases expression of serine/threonine-protein kinase 3 (RIOK3), wherein the ribonucleotide agent comprises:
- an antisense oligonucleotide, short hairpin RNA (shRNA), small interfering RNA (siRNA), an asymmetrical iRNA (aiRNA), a microRNA, a miniRNA, a lncRNA, or a ribozyme, or any combination thereof;
- and
- a forward sequence comprising the nucleotide sequence of SEQ ID NO:
- 3, or a reverse sequence comprising the nucleotide sequence of SEQ ID NO:
- 4, or both,
- wherein the ribonucleotide agent targets the mRNA sequence of SEQ ID NO:
- 1, or SEQ ID NO:
- 2, or both.
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An isolated polynucleotide comprising the nucleic acid sequence of SEQ ID NO:
- 3, or the nucleic acid sequence of SEQ ID NO:
- 4, or both.
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Dependencies are inferred from the claim text.