A novel method to increase transgene expression and the stability of gene therapy-associated episomal vectors

Non-viral episomal vectors offer a safe and attractive alternative to viral and integrated vectors by avoiding insertional mutagenesis and position effects, making them ideal expression vectors for gene therapy [1].The first non-viral episomal vector, pEPI-1, which is based on the full-length scaffold/matrix attachment region (S/MAR), was established by Piechaczek et al. [2].The full-length S/MAR element interacts with the nuclear matrix via the matrix protein SAF-A, thereby maintaining mitotic stability and transgene expression [3,4].Several strategies, including optimization of

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Publication Details

Journal
Acta Biochimica et Biophysica Sinica
Published
2025-06-16
DOI
https://doi.org/10.3724/abbs.2025104
Primary Topic
Virus-based gene therapy research
Type
article
Field-Weighted Citation Impact
0.00

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A novel method to increase transgene expression and the stability of gene therapy-associated episomal vectors

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Acta Biochimica et Biophysica Sinica
Virus-based gene therapy research
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A novel method to increase transgene expression and the stability of gene therapy-associated episomal vectors

Yan‐Long Jia, Mengying Ji, Tianyun Wang, Rui Liu, Xi Zhang, Zihan Guo, Wen Wang, Zimeng Han, Xiao-Yin Wang
article en

Abstract

Non-viral episomal vectors offer a safe and attractive alternative to viral and integrated vectors by avoiding insertional mutagenesis and position effects, making them ideal expression vectors for gene therapy [1].The first non-viral episomal vector, pEPI-1, which is based on the full-length scaffold/matrix attachment region (S/MAR), was established by Piechaczek et al. [2].The full-length S/MAR element interacts with the nuclear matrix via the matrix protein SAF-A, thereby maintaining mitotic stability and transgene expression [3,4].Several strategies, including optimization of

Acta Biochimica et Biophysica Sinica
Henan Medical University (CN)
National Natural Science Foundation of China, Natural Science Foundation of Henan Province, Henan Provincial Science and Technology Research Project
Zero hunger
Openalex Percentile: Top 84%
Virus-based gene therapy research
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