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US 11,590,085 B2Granted
Compositions and methods for organ specific delivery of nucleic acids
AssigneeBoard of Regents, The University of Texas System
InventorsQiang Cheng, Tuo Wei, Daniel J. Siegwart
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Abstract
The present disclosure provides compositions which shown preferential targeting or delivery of a nucleic acid composition to a particular organ. In some embodiments, the composition comprises a steroid or sterol, an ionizable cationic lipid, a phospholipid, a PEG lipid, and a permanently cationic lipid which may be used to deliver a nucleic acid.
Claims30
A method for targeted delivery of a gene or transcript editing composition to an organ or a cell therein, the method comprising:
- contacting said organ or said cell therein with said gene or transcript editing composition assembled with a selective organ targeting (SORT) lipid composition,
- wherein said SORT lipid composition comprises:
- (1) an ionizable cationic lipid;
- and (2) a SORT lipid selected from the group consisting of a cationic SORT lipid, a zwitterionic SORT lipid, and an anionic SORT lipid;
- thereby providing a modified expression profile of a target gene or transcript in said organ or said cell therein as compared to that achieved absent said SORT lipid.
2 more elements
Dependencies are inferred from the claim text.