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US 11,767,529 B2Granted
Short hairpin RNA compositions, methods of making and applications thereof
AssigneeCity of Hope
InventorGuihua Sun
Life of this patent
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Abstract
Provided herein are novel synthetic short hairpin RNA (shRNA) molecules and compositions and kits comprising such molecules, as well as methods of making and using these molecules, compositions, and kits.
Claims13
A synthetic short hairpin RNA (shRNA) molecule designed to silence the expression of a target gene, the synthetic shRNA molecule comprising:
- (a) a 5′ arm and a 3′ arm comprising:
- (i) a stem region comprising 16, 17 or 18 base pairs, the base pairs comprising nucleotides from the 5′ arm paired with nucleotides from the 3′ arm, and
- (ii) one or more unpaired nucleotides at the 5′ terminal end of the 5′ arm and an unpaired dideoxycytidine nucleotide (ddC) at the 3′ terminal end of the 3′ arm;
- and
- (b) a loop region comprising 4 nucleotides that connects the 5′ arm to the 3′ arm,
- wherein the synthetic shRNA molecule is processed by Argonaute 2 (Ago2) in a Dicer-independent manner.
3 more elements
A method of silencing expression of a target gene comprising:
- obtaining a sample comprising the target gene, and
- providing a synthetic short hairpin RNA (shRNA) molecule designed to silence the expression of the target gene, wherein the shRNA molecule comprises:
- (a) a 5′ arm and a 3′ arm comprising:
- (i) a stem region comprising 16, 17 or 18 base pairs, the base pairs comprising nucleotides from the 5′ arm paired with nucleotides from the 3′ arm, and
- (ii) one or more unpaired nucleotides at the 5′ terminal end of the 5′ arm and an unpaired dideoxycytidine nucleotide (ddC) at the 3′ terminal end of the 3′ arm;
- and
- (b) a loop region comprising 4 nucleotides that connects the 5′ arm to the 3′ arm,
- wherein the synthetic shRNA molecule is processed by Argonaute 2 (Ago2) in a Dicer-independent manner.
5 more elements
Dependencies are inferred from the claim text.