Polycationic sterol derivatives as transfection agents
AssigneeImperial College of Science, Technology and Medicine
InventorsAndrew Miller, Robert Cooper, Christopher Etheridge
Life of this patent
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Abstract
A method for treating genetic disorders or conditions or diseases in patients in need of such treatments. The method includes administering an effective amount of a compound comprising a cholesterol group or derivative thereof having linked thereto a head group. The head group is more positive than the heat group of DC-Chol. The heat group may be a straight chain polymeric group having two or more amine groups separated by an ethylene group.
Claims50
A compound capable of acting as a cationic lipid, the compound comprising a cholesterol group having linked thereto a head group;
- and wherein the head troup is more positive than the head group of DC-Chol;
- but wherein the compound is not synthesised by reacting spermidine and cholesterol chloroformate in CH2Cl2 in the presence of N,N-diisoprophylethylamine.
2-22. (canceled)
A method for treating a genetic disorder, or condition or disease in a patient in need of treatment, comprising:
- administering an effective amount of a compound comprising a cholesterol group or derivative thereof having linked thereto a head group;
- wherein the head group is more positive than the head group of DC-Chol;
- further wherein the head group is a straight chain polyamine;
- further wherein two or more of the amine groups of the polyamine group are separated by an ethylene group.
A method for treating a genetic disorder, or condition or disease in a patient in need of treatment, comprising:
- administering an effective amount of a compound selected from the group consisting of cationic lipid compounds, cationic liposomes formed from cationic lipid compounds, cationic lipid compounds in admixture with or associated with a nucleotide sequence, cationic liposomes, formed from a cationic lipid compound, in admixture with or associated with a nucleotide sequence, and combinations thereof,
- the compound comprising a cholesterol group or derivative thereof having linked thereto a head group;
- wherein the head group is more positive than the head group of DC-Choi;
- further wherein the head group is a straight chain polyamine;
- further wherein two or more of the amine groups of the polyamine group are separated by an ethylene group.
A method for treating a genetic disorder, or condition or disease in a patient in need of treatment, comprising:
- administering an effective amount of a composition, the composition comprising:
- i. a compound selected from the group consisting of cationic lipid compounds, cationic liposomes formed from a cationic lipid compound, and combinations thereof, the compound comprising a cholesterol group or derivative thereof having linked thereto a head group;
- wherein the head group is more positive than the head group of DC-Chol;
- further wherein the head group is a straight chain polyamine;
- further wherein two or more of the amine groups of the polyamine group are separated by an ethylene group, and
- ii. a pharmaceutical, and optionally a pharmaceutically acceptable diluent, carrier or excipient.
A method for treating a genetic disorder or condition or disease in a patient in need of treatment, comprising:
- administering an effective amount of a composition comprising a compound selected from the group consisting of cationic lipid compounds, cationic lipid compounds in admixture with or associated with a nucleotide sequence, cationic liposomes (formed from a cationic lipid compound) in admixture with or associated with a nucleotide sequence, and combinations thereof;
- the compound comprising a cholesterol group or derivative thereof having linked thereto a head group;
- wherein the head group is more positive than the head group of DC-Chol;
- further wherein the head group is a straight chain polyamine;
- further wherein two or more of the amine groups of the polyamine group are separated by an ethylene group.
A method for the treatment of a genetic disorder or condition or disease in a patient in need thereof, comprising administering a cationic lipid compound, the compound comprising a cholesterol group or derivative thereof having linked thereto a head group, wherein the head group is more positive than the head group of DC-Chol;
- further wherein the head group is a polyamine group which is a straight chain polyamine group;
- further wherein two or more of the amine groups of the polyamine group are separated by an ethylene group.
A method for the treatment of a genetic disorder or condition or disease in a patient in need thereof, comprising administering a cationic liposome formed from a cationic lipid compound, the compound comprising a cholesterol group having linked thereto a head group;
- wherein the head group is more positive than the head group of DC-Chol;
- further wherein the head group is a polyamine group which is a straight chain polyamine group;
- further wherein two or more of the amine groups of the polyamine group are separated by an ethylene group.
A method for the treatment of a genetic disorder or condition or disease in a patient in need thereof, comprising administering a cationic lipid compound in admixture with or associated with a nucleotide sequence, the compound comprising a cholesterol group or derivative thereof having linked thereto a head group;
- wherein the head group is more positive than the head group of DC-Chol;
- further wherein the head group is a polyamine group which is a straight chain polyamine group;
- further wherein two or more of the amine groups of the polyamine group are separated by an ethylene group.
A method for the treatment of a genetic disorder or condition or disease in a patient in need thereof, comprising administering a cationic liposome in admixture with or associated with a nucleotide sequence, wherein the cationic liposome is formed from a cationic lipid compound, the compound comprising a cholesterol group having linked thereto a head group;
- wherein the head group is more positive than the head group of DC-Chol;
- further wherein the head group is a polyamine group which is a straight chain polyamine group;
- further wherein two or more of the amine groups of the polyamine group are separated by an ethylene group.
A method for the treatment of a genetic disorder or condition or disease in a patient in need thereof, comprising administering a pharmaceutical composition comprising
- (i) a cationic lipid compound, the compound comprising a cholesterol group or derivative thereof having linked thereto a head group;
- wherein the head group is more positive than the head group of DC-Chol;
- further wherein the head group is a polyamine group which is a straight chain polyamine group;
- further wherein two or more of the amine groups of the polyamine group are separated by an ethylene group;
- and
- (ii) a pharmaceutical and, optionally, a pharmaceutically acceptable diluent, carrier or excipient.
A method for treatment of a genetic disorder or condition or disease in a patient in need thereof, comprising administering a pharmaceutical composition comprising:
- (i) a cationic liposome formed from a cationic lipid compound, the compound comprising a cholesterol group having linked thereto a head group;
- wherein the head group is more positive than the head group of DC-Chol;
- further wherein the head group is a polyamine group which is a straight chain polyamine group;
- further wherein two or more of the amine groups of the polyamine group are separated by an ethylene group;
- and
- (ii) a pharmaceutical and, optionally, a pharmaceutically acceptable diluent, carrier or excipient.
Dependencies are inferred from the claim text.