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US 2023/0407308 A1Published application
Conditional-sirnas and uses thereof in treating acute myeloid leukemia
AssigneesCity of Hope, California Institute of Technology
InventorsGuido MARCUCCI, Ya-Huei KUO, Si-Ping HAN, Lisa SCHERER, William A. GODDARD, III, John ROSSI
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Abstract
Disclosed herein are conditional siRNAs activatable by CBFβ-MYH11 oncogenic gene and use thereof for treating conditions such as acute myeloid leukemia (AML). The conditional siRNAs target MCL-1 or HDAC8.
Claims23
A conditional RNA-sensor complex comprising:
- a sensor strand comprising at least one toehold segment, wherein the toehold segment binds a pathological biomarker present in or overexpressed in a target cell;
- and
- a double stranded pro-siRNA molecule comprising
- a guide strand comprising an RNA molecule that binds a therapeutic target molecule in the target cell;
- and
- a core strand comprising
- a first portion comprising a passenger strand that is fully or partially complimentary to and binds the guide strand;
- a second portion comprising a first protection segment that is fully or partially complimentary to and binds the sensor strand;
- and
- a first linker that joins a first end of the passenger strand to the first protection segment.
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Dependencies are inferred from the claim text.