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US 2019/0240340 A1Published application
Functionalized nucleic acid nanostructures for rna delivery
AssigneeGuild BioSciences
InventorsIan Fleming, Xi Chun Zhou
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Abstract
The present disclosure provides cell-penetrating nucleic acid nanostructures well suited as transfection reagents for the delivery of bioactive agents to cells both in vivo and in vitro for research, diagnostic, and/or therapeutic purposes.
Claims17
A nucleic acid nanostructure, comprising:
- a DNA or RNA scaffold;
- at least one single stranded nucleic acid staple strand complementary to the scaffold;
- at least one cell penetrating peptide attached to the at least one staple strand;
- and
- at least one therapeutic substance attached to the at least one staple strand;
- wherein the at least one cell penetrating peptide is positively charged,
- wherein the at least one therapeutic agent is selected from the group:
- siRNA, miRNA, shRNA, asRNA, mRNA, crRNA, tracrRNA, and a RNA vaccine.
5 more elements
(canceled)
A method of treating a subject, the method comprising:
- providing a nucleic acid nanostructure having:
- a DNA or RNA scaffold;
- at least one single-stranded nucleic acid staple strand complementary to the scaffold;
- at least one positively charged cell penetrating peptide attached to the at least one staple strand;
- and
- at least one therapeutic substance attached to the at least one staple strand;
- wherein the at least one therapeutic agent is selected from the group:
- siRNA, miRNA, shRNA, asRNA, mRNA, crRNA, tracrRNA, or a RNA vaccine;
- administering a therapeutically effective amount of the nucleic acid nanostructure to a subject.
6 more elements
(canceled)
Dependencies are inferred from the claim text.