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US 11,974,969 B2Granted
Artificially synthesized sphingosine derivative lipoid monomer and use of same for delivering nucleic acid
AssigneeInstitute of Basic Medical Sciences, Chinese Academy of Medical Sciences
InventorsChengyu Jiang, Yuhao Qin, Xiaoyun Li, Cong Zhang
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Abstract
The invention provides an artificially synthesized single sphingosine lipid and use of delivering a nucleic acid thereof. More particularly, the invention provides Use or method for delivering a nucleic acid to a cell or a subject using a compound of Formula (I), a stereoisomer or a pharmaceutical acceptable salt thereof, or a combination comprising a compound of Formula (I), a stereoisomer or a pharmaceutical acceptable salt thereof,
Claims17
A method for delivering a nucleic acid to a cell or a subject, comprising:
- (1) providing an organic solvent solution comprising a lipid composition;
- (2) providing an aqueous solution comprising the nucleic acid;
- (3) adding the organic solvent solution of step (1) to the aqueous solution of step (2), and then heating at a temperature of 60° C. to 100° C. to obtain a lipid-nucleic acid mixture, and
- (4) contacting the lipid-nucleic acid mixture of step (3) with the cell or the subject so that the nucleic acid is delivered to the cell or the subject,
- wherein the lipid composition consists of one or more compounds of Formula (I), a stereoisomer or a pharmaceutical acceptable salt thereof:
- wherein:
- R1 is selected from C1-20 alkyl or C2-20 alkenyl, which are optionally substituted by one to three hydroxyl groups;
- R2 is hydrogen and R3 is hydroxyl;
- or
- R2 and R3 together form oxo (═O);
- R4 and R5 are independently selected from the group consisting of hydrogen, C1-20 alkyl, C1-30 alkylacyl and C1-30 alkenylacyl, said C1-30 alkylacyl and C1-30 alkenylacyl are optionally substituted by one to three groups selected from biotin, acyl or hydroxyl;
- R6 is selected from the group consisting of hydrogen, hydroxyl, phosphate ester group, —O-glycosyl, ganglioside, and aminoethoxyphosphonate ester group (NH2—CH2—CH2—O—P(O)OH—).
9 more elements
Dependencies are inferred from the claim text.