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US 10,894,960 B2Granted
Compositions and methods for nucleic acid transfer
AssigneeChildren's Hospital Medical Center
InventorsAssem G. Ziady, Matthew Siefert, Songbai Lin, Harrison Brown
Life of this patent
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Abstract
Disclosed are methods for the enhancement of nucleic acid delivery systems. The methods may employ treatment with a compound and/or an RNAi molecule in combination with a nucleic acid to improve nucleic acid uptake into a cell. In particular, the disclosed methods may be useful for improved gene therapy techniques.
Claims14
A method for enhancing uptake of a nucleic acid nanoparticle into a eukaryotic cell for administration of a therapeutic gene, comprising
- a. contacting said eukaryotic cell with said nucleic acid nanoparticle (NNP);
- and
- b. contacting said eukaryotic cell with a nanoparticle uptake enhancing agent selected from one or both of an RNAi molecule and a compound;
- wherein said NNP comprises a nucleic acid encoding said therapeutic gene or active portion thereof, and a cationic polymer conjugated to polyethylene glycol;
- wherein said cationic polymer is selected from a poly-lysine, a polyethyleneimine, and combinations thereof;
- wherein RNAi molecule inhibits expression of a gene encoding a protein selected from keratin 13, APC protein, protocadherin 17, and spectrin alpha;
- and
- wherein said compound is selected from geldanamycin, alvespimycin, entasobulin, androstanolone, spermine, cortisone, quercetin, acetohexamide, resveratrol, doxorubicin, ruxolitinib, roscovitine, sildenafil, teniposide, etoposide, 3-Aminobenzamide (INO 1001), diazoxide, tedisamil, glimepiride, epirubicin, annamycin, repaglinide, 4β-aminoalkyl-4′-O-demethyl-4-desoxypodophyllotoxin (TOP53), acetohexamide, elsamitrucin, ketamine, NK109, tifenazoxide, olaparib, intoplicine, and combinations thereof, As2O3.
5 more elements
A method of treating an individual, comprising the step of
- a. administering an RNAi that inhibits expression of one or more genes encoding a protein selected from keratin 13, APC protein, protocadherin 17, spectrin alpha, and combinations thereof, and a compound selected from geldanamycin, alvespimycin, entasobulin, androstanolone, spermine, cortisone, quercetin, acetohexamide, resveratrol, doxorubicin, ruxolitinib, roscovitine, sildenafil, teniposide, etoposide, 3-Aminobenzamide (INO 1001), diazoxide, tedisamil, glimepiride, epirubicin, annamycin, repaglinide, 4β-aminoalkyl-4′-O-demethyl-4-desoxypodophyllotoxin (TOP53), acetohexamide, elsamitrucin, ketamine, NK109, tifenazoxide, olaparib, intoplicine, As2O3;
- and
- b. administering a nucleic acid nanoparticle (NNP) comprising a therapeutic gene, or portion of a therapeutic gene that encodes an active portion of a protein, that is expressed in said individual to provide a functional protein or functional protein fragment, wherein said functional protein or functional protein fragment is expressed in said individual in a therapeutically effective amount;
- wherein said NNP comprises a cationic polymer conjugated to polyethylene glycol and;
- wherein said cationic polymer is selected from a poly lysine, a polyethyleneimine, and combinations thereof.
2 more elements
Dependencies are inferred from the claim text.