Compositions and methods for the delivery of nucleic acids
AssigneeCase Western Reserve University
InventorsZheng-Rong Lu, Da Sun, Rebecca Schur
Life of this patent
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Abstract
A nanosized complex includes a nucleic acid and a compound comprising formula (I): wherein R1 is an alkylamino group or a group containing at least one aromatic group; R2 and R3 are independently an aliphatic group or hydrophobic group; R4 and R5 are independently H, a substituted or unsubstituted alkyl group, an alkenyl group, an acyl group, or an aromatic group, or a polymer, a targeting group, a detectable moiety, or a linker, or a combination thereof, and at least one of R4 and R5 includes a retinoid or retinoid derivative that targets and/or binds to an interphotoreceptor retinoid binding protein; a, b, c, and d are independently an integer from 1 to 10; and pharmaceutically acceptable salts thereof.
Claims21
A nanosized complex comprising:
- a nucleic acid that is complexed with a compound, the compound comprising formula (I):
- wherein R1 is an alkylamino group or a group containing at least one aromatic group;
- R2 and R3 are independently an aliphatic group or a hydrophobic group;
- R4 and R5 are independently H, a substituted or unsubstituted alkyl group, an alkenyl group, an acyl group, or an aromatic group, or a polymer, a targeting group, a detectable moiety, or a linker, or a combination thereof, and at least one of R4 and R5 is a retinoid or retinoid derivative, each with an optional linker, that targets and/or binds to an interphotoreceptor retinoid binding protein;
- a, b, c, and d are independently an integer from 1 to 10;
- and pharmaceutically acceptable salts thereof.
A method of treating a retinal disorder in a subject in need thereof, the method comprising:
- administering to the subject a nanosized complex comprising:
- a nucleic acid that is complexed with a compound, wherein the nucleic acid is capable of treating, ameliorating, attenuating, and/or eliminating symptoms of the retinal disorder when the complex is introduced to or within an eye of a subject, and wherein the compound comprises formula (I):
- wherein R1 is an alkylamino group or a group containing at least one aromatic group;
- R2 and R3 are independently an aliphatic group or a hydrophobic group;
- R4 and R5 are independently H, a substituted or unsubstituted alkyl group, an alkenyl group, an acyl group, or an aromatic group, or a polymer, a targeting group, a detectable moiety, or a linker, or a combination thereof, and at least one of R4 and R5 is a retinoid or retinoid derivative, each with an optional linker, that targets and/or binds to an interphotoreceptor retinoid binding protein;
- a, b, c, and d are independently an integer from 1 to 10;
- and pharmaceutically acceptable salts thereof.
Dependencies are inferred from the claim text.