COMPOSITIONS AND METHODS FOR ENHANCING DELIVERY OF THERAPEUTIC AGENTS TO CELLS
First Claim
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1. A method of delivering an agent to cells, the method comprising administering the agent to the cells in a composition comprising a delivery enhancing compound of Formula I:
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wherein;
m and n are the same or different and each is an integer from 2-8;
R is a cationic group or X1 is a cholic acid group or deoxycholic acid group; and
X2 and X3 are each independently selected from the group consisting of a cholic acid group, a deoxycholic acid group, and a saccharide group;
wherein at least one of X2 and X3 is a saccharide group when R is
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Abstract
This invention provides methods and compositions for enhancing transfer of an agent into a cell. The agents can include polypeptides, polynucleotides such as genes and antisense nucleic acids, and other molecules. In some embodiments, the agents are modulating agents that can modulate a cellular activity or function when introduced into the cell. The methods and compositions are useful for introducing agents into individual cells, as well as cells that are present as a tissue or organ.
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Citations
60 Claims
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1. A method of delivering an agent to cells, the method comprising administering the agent to the cells in a composition comprising a delivery enhancing compound of Formula I:
-
wherein;
m and n are the same or different and each is an integer from 2-8;
R is a cationic group orX1 is a cholic acid group or deoxycholic acid group; and
X2 and X3 are each independently selected from the group consisting of a cholic acid group, a deoxycholic acid group, and a saccharide group;
wherein at least one of X2 and X3 is a saccharide group when R is - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24, 25, 26, 27)
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28. A composition for delivering an agent to cells, the composition comprising the agent and a delivery enhancing compound of Formula I:
-
wherein;
m and n are the same or different and each is an integer from 2-8;
R is a cationic group orX1 is a cholic acid group or deoxycholic acid group; and
X2 and X3 are each independently selected from the group consisting of a cholic acid group, a deoxycholic acid group, and a saccharide group;
wherein at least one of X2 and X3 is a saccharide group when R is - View Dependent Claims (29, 30, 31, 32, 33, 34, 35, 36, 37, 38, 39, 40, 41)
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42. A delivery enhancing compound having a Formula I:
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wherein;
m and n are the same or different and each is an integer from 2-8;
R is a cationic group orX1 is a cholic acid group or deoxycholic acid group; and
X2 and X3 are each independently selected from the group consisting of a cholic acid group, a deoxycholic acid group, and a saccharide group;
wherein at least one of X2 and X3 is a saccharide group when R is - View Dependent Claims (43, 44, 45, 46, 47, 48, 49, 50, 51, 52, 53, 54, 55, 56, 57, 58)
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59. A delivery enhancing compound of Formula II:
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wherein X1 and X2 are selected from the group consisting of a cholic acid group and a deoxycholic acid group and X3 is a saccharide group. - View Dependent Claims (60)
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Specification