Nucleic acid-controlled catalytic rnas for trigger-responsive regulation
US-2024425855-A1 · Dec 26, 2024 · US
US11208653B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-11208653-B2 |
| Application number | US-201716097179-A |
| Country | US |
| Kind code | B2 |
| Filing date | Apr 27, 2017 |
| Priority date | Apr 27, 2016 |
| Publication date | Dec 28, 2021 |
| Grant date | Dec 28, 2021 |
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Methods and compositions for increasing RNAi efficiency through single nucleotide mismatches.
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What is claimed is: 1. A method of treating a mammal suffering from a condition, the method comprising administering to the mammal a therapeutically effective amount of an siRNA targeted to a target gene having a target sequence, wherein the siRNA comprises a guide sequence having at least one mismatch with respect to the target sequence, wherein the guide sequence mismatch with respect to the target sequence is located at guide position 6 of the guide sequence and is a G-G mismatch. 2. The method of claim 1 , wherein the siRNA is administered parenterally. 3. The method of claim 1 , wherein the siRNA is administered intravenously. 4. The method of claim 1 , wherein the siRNA is administered by bolus injection into a target organ or tissue. 5. The method of claim 1 , wherein the siRNA is administered intraperitoneally. 6. The method of claim 1 , wherein the siRNA is administered subcutaneously. 7. The method of claim 1 , wherein the mammal is a human.
interfering nucleic acids [NA] · CPC title
General methods applicable to biologically active non-coding nucleic acids · CPC title
Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; {Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing (when used in plants C12N15/8218)} · CPC title
Methods for regulating/modulating their activity · CPC title
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