Use of compacted nucleic acid nanoparticles in non-viral treatments of ocular diseases
First Claim
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1. A method of treating a subject having an ocular disorder, comprising:
- providing a non-viral nanoparticle comprising a compacted nucleic acid molecule linked to a polymer component, wherein the nucleic acid molecule is DNA or RNA and wherein the non-viral nanoparticle has a minor diameter which is less than 25 nm; and
administering the non-viral nanoparticle to an eye of the subject in a manner such that the nucleic acid molecule is transfected within an ocular cell of the eye of the subject and is able to be expressed therein.
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Abstract
The present invention is a method of using compacted nucleic acid (such as DNA) nanoparticles for non-viral gene transfer to various tissues of the human eye or eyes of other mammals. These nanoparticles comprise, in one embodiment, a neutrally-charged complex containing a single molecule of plasmid DNA compacted with polyethylene glycol (PEG)-substituted poly lysine peptides.
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14 Claims
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1. A method of treating a subject having an ocular disorder, comprising:
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providing a non-viral nanoparticle comprising a compacted nucleic acid molecule linked to a polymer component, wherein the nucleic acid molecule is DNA or RNA and wherein the non-viral nanoparticle has a minor diameter which is less than 25 nm; and administering the non-viral nanoparticle to an eye of the subject in a manner such that the nucleic acid molecule is transfected within an ocular cell of the eye of the subject and is able to be expressed therein. - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12)
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13. A compacted nanoparticle, comprising:
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a nucleic acid linked to a polymer component, the nucleic acid comprising at least one of CA4, CRX, FSCN2, GUCA1B, IMPDH1, NR2E3, NRL, PRPF3, PRPF8, PRPF31, PRPH2, RHO, ROM1, RP1, RP9, SEMA4A, TOPORS, ABCA4, CERKL, CNGA1, CNGB1, CRB1, LRAT, MERTK, NRL, PDE6A, PDE6B, PRCD, PROM1, RGR, RLBP1, RP1, RPE65, SAG, TULP1, USH2A, RP2, RPGR, ABCA4, ARMS2, C2, C3, CFB, CFH, ERCC6, FBLN5, HMCN1, HTRA1, RAX2, TLR4, BEST1, C1QTNF5, EFEMP1, ELOVL4, FSCN2, GUCA1B, PRPH2, TIMP3, and RPGR; and a promoter sequence; and wherein the nanoparticle has a minor diameter of less than 25 nm, and wherein the polymer component comprises a PEG molecule covalently linked to a peptide component comprising a terminal cysteine molecule and multiple repeating lysine or arginine residues. - View Dependent Claims (14)
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Specification