Nonviral gene transfer to the suprachoroidal space
AssigneeThe Johns Hopkins University
InventorsPeter A. Campochiaro, Jordan Green, Jayoung Kim, Jikui Shen
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Abstract
The presently disclosed subject matter provides compositions and methods for administering a nanoparticle or microparticle and a therapeutic agent to the suprachoroidal space in the eye.
Claims18
A method for treating a disease or condition of the eye, the method comprising non-virally transfecting one or more cells in a suprachoroidal space by administering to a suprachoroidal space of an eye of a subject a composition comprising a nanoparticle or a microparticle comprising a poly(beta-amino ester) (PBAE) and a therapeutic agent, wherein the therapeutic agent is selected from an expression construct, a therapeutic transgene, a therapeutic protein, a viral vector, a plasmid, a gene, a nucleic acid, DNA, RNA, and mRNA, and wherein the PBAE is
- wherein:
- n is an integer from 1 to 10,000;
- R1, R2, R3, R4, R5, R6, R7, R8, and R9 are each hydrogen;
- R1 is absent and when present the compound of formula (I) further comprises a counter ion selected from the group consisting of chloride, fluoride, bromide, iodide, sulfate, nitrate, fumarate, acetate, carbonate, stearate, laurate, and oleate;
- and
Dependencies are inferred from the claim text.