Supercoiled minicircle dna for gene therapy applications

摘要

Disclosed is a nucleic acid molecule composition comprising a minivector, wherein said minivector lacks both a bacterial origin or replication and an antibiotic selection gene, wherein the minivector has a size up to 1,200 base pair and wherein the minivector encodes a nucleic acid sequence (e.g. short hair pin RNA, shRNA, microRNA, miRNA). It further discloses the methods of silencing gene expression (anaplastic lymphoma kinase gene), treating cancer, gene expression and gene therapy by contacting the minivector to an isolated cell.