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US 12,674,164 B2Granted
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.
Claims19
A conditional RNA-sensor complex comprising:
- a sensor strand comprising at least one toehold segment, wherein the toehold segment binds a pathological biomarker that is associated with acute myeloid leukemia (AML) and present in or overexpressed in a target cell, wherein the sensor strand comprises a sequence having at least 95% homology to SEQ ID NO:1;
- 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, wherein the therapeutic target molecule is MCL-1 or HDAC8;
- 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.
7 more elements
Dependencies are inferred from the claim text.