Advances of Non-Viral Episomal Vectors for Gene Therapy
ZHANG Weili1, DU Qiujie1, ZHU Jiayi1, WANG Haomin1, QIAO Kexin1, WANG Tianyun1,2, ZHAO Chunpeng1 *, ZHANG Xi2,3, WANG Xiaoyin1,2*
Gene transfer vector plays a key role in gene therapy. Non-viral episomal vectors can be episomal replication within the host cell rather than be integrated to the host to overcome the adverse effects of viral vector and integrated vector, so it is a safe and ideal expression vector for gene therapy. However, its application in gene therapy is limited because of its low clone formation rate, low expression and low copy number. Since the construction of the first generation of non-viral episomal vectors, a series of measures, such as truncating MAR elements, reducing the number of CpG motifs, selecting suitable promoters, using regulatory elements have been used to regulate their expression levels and stability, resulting in improving transfection efficiency, and make it exhibit higher expression level and stability. And these vectors have been widely used in gene therapy research for various systemic diseases. This paper reviewed the research progress of various strategies for optimization of non-viral episomal vectors and discussed their applications in gene therapy.
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