Therapeutic nanospheres
AssigneeThe Johns Hopkins University
InventorsScott Walsh, Ronald Rubenstein, Pam Zeitlin, Kam W. Leong
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Abstract
4-Phenylbutyrate exerts many beneficial biological effects. It appears to induce the transcription of certain promoters, as well as having a remedial effect on proteins which are aberrantly localized within the cell. In addition, it appears to cause cells to developmentally differentiate. The present invention provides nanosphere formulations of 4-phenylbutyrate and other drugs which remediate defective protein localization intracellularly. These formulations permit lower concentrations of drugs to be administered, providing both cost and safety benefits.
Claims19
A solid nanosphere of comprising sodium 4-phenylbutrate (4-PBA) wherein the nanosphere is formed by coacervation of a polycation and a polyanion.
A solid nanosphere comprising:
- the wild-type CFTR-encoding nucleic acid;
- and
- a drug which activates ΔF508 mutant CFTR proteins.
A solid nanosphere comprising:
- sodium 4-phenylbutyrate (4-PBA) and a nucleic acid construct, wherein the construct comprises a promoter operatively linked to a gene coding sequence, wherein the promoter is 4-PBA-inducible.
Dependencies are inferred from the claim text.