BLOCK COPOLYMERS AND THEIR CONJUGATES OR COMPLEXES WITH OLIGONUCLEOTIDES

Described herein are block copolymers, and methods of making and utilizing such copolymers. The described block copolymers are disruptive of a cellular membrane, including an extracellular membrane, an intracellular membrane, a vesicle, an organelle, an endosome, a liposome, or a red blood cell. Pre...

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Bibliographische Detailangaben
Hauptverfasser: HOUSTON, Michael E. Jr, JOHNSON, Russell N, MONAHAN, Sean D, PASCHAL, Amber E, HARVIE, Pierrot, DECLUE, Michael S, QABAR, Maher, PRIEVE, Mary G, ROY, Debashish, DIAB, Charbel, GALPERIN, Anna
Format: Patent
Sprache:eng
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Zusammenfassung:Described herein are block copolymers, and methods of making and utilizing such copolymers. The described block copolymers are disruptive of a cellular membrane, including an extracellular membrane, an intracellular membrane, a vesicle, an organelle, an endosome, a liposome, or a red blood cell. Preferably, in certain instances, the block copolymer disrupts the membrane and enters the intracellular environment. In specific examples, the block copolymer is endosomolytic and capable of delivering an oligonucleotide (e.g., an mRNA) to a cell. Compositions comprising a block copolymer and an oligonucleotide (e.g., an mRNA) are also disclosed. WO 2015/017519 PCT/US2014/048839 1.0- A AA z A o* .. *.. 0.5 * ccNoME Timepoint 24hr 48hr 72hr 96hr 6d 10d 72hr 10d at Sacrifice 3 mg/kg p-catenin siRNA Buffer