Compounds promoting delivery of genes
AssigneeThe UAB Research Foundation
InventorEric Sorscher
Life of this patent
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Abstract
A process is provided for activating gene transfer in a cell by administering a gene transfer activating compound to the cell in conjunction with a gene transfer vector. A kit for activating gene transfer as well as process for identifying a compound that activates gene transfer are described. Use of a gene transfer activator compound enhances gene transfer of a given gene transfer vector.
Claims40
A kit for activating gene transfer, said kit comprising a gene transfer activating compound, packaged in a suitable container together with instructions for use to activate gene transfer.
18-27. (canceled)
A process for activating gene transfer of a vector to a cell comprising the steps of:
- contacting a cell with a recombinant gene transfer vector:
- and
- administering a gene transfer activating compound to the cell, such that transfer of the vector to the cell is activated;
- wherein the gene transfer activating compound is selected from the group consisting of:
- wherein Q is nitrogen or oxygen, wherein each occurrence of R1 independently is H, CH3, CH2CH3 or a nullity, wherein R2 is C1-C18 alleyl, C2-C18 ether, C2-C18 thioether, C2-C18 secondary or tertiary amine,
- wherein A is
- wherein R3 is H, C1-C6 alkyl, or a heteroatom substituted C1-C6 alkyl where the heteroatom is oxygen, nitrogen, or sulfur, wherein R4 is C2-C6 amide, or ═N-R5 where R5 is C7-C12 aryloxyl, C1-C6 hydronyl, carbonyl, carboxyl, or acyl, imidazyl, pyrazyl, thiazyl, or oxazyl, wherein X is H, F, Cl or Br, wherein Z is oxygen or sulfur.
A process for activating gene transfer of a vector to a cell comprising the steps of:
- contacting a cell with a recombinant gene transfer vector;
- and
- administering a gene transfer activating compound to the cell, such that transfer of the vector to the cell is activated;
- wherein the gene transfer activating compound is selected from the group consisting of:
- NSC73609, NSC82090, NSC101492, NSC102821, NSC106191, NSC108613, NSC109325, NSC128720, NSC143491, NSC259968, NSC373989 and NSC675865.
30-35. (canceled)
A process for determining the efficacy of a putative gene transfer activating compound to activate gene transfer, comprising the steps of:
- administering a test compound to a first cell;
- contacting the first cell with a first amount of a recombinant vector;
- contacting a second cell with a second amount of the recombinant vector, the second amount of the recombinant vector substantially equal to the first amount;
- measuring a gene transfer indicator in the first cell to obtain a test measurement;
- measuring the gene transfer indicator in the second cell to obtain a control measurement;
- and
- comparing the test measurement and the control measurement to determine the efficacy of the putative gene transfer activating compound to activate gene transfer.
Use of a compound of Formulae I-VII for use as a gene transfer activating compound.
(canceled)
Dependencies are inferred from the claim text.