Modified closed-loop DNA (ceDNA) containing symmetrical modified inverted repeats

摘要

Described herein are ceDNA vectors with linear and continuous structures that can be produced in high yield and used for efficient delivery and expression of transgenes. According to some embodiments, the ceDNA vector comprises at least one heterologous nucleotide sequence operably disposed between two flanking symmetric inverted repeat sequences that are not wild-type AAV ITRs, wherein all or some of the heterologous nucleotide sequences are at least It is under the control of one control switch. Some ceDNA vectors provided herein further comprise cis-regulatory elements and provide high gene expression efficiency. Furthermore, presented herein are methods and cell lines for the reliable and efficient production of linear, continuous and capsid-free DNA vectors.