Engineering Nanomedicine for Non-Viral RNA-Based Gene Therapy of Glioblastoma

Wenya He; Ningyang Wang; Yaping Wang; Mengyao Liu; Qian Qing; Qihang Su; Yan Zou; Yang Liu

doi:10.3390/pharmaceutics16040482

Engineering Nanomedicine for Non-Viral RNA-Based Gene Therapy of Glioblastoma

Pharmaceutics. 2024 Apr 1;16(4):482. doi: 10.3390/pharmaceutics16040482.

Authors

Wenya He^{1

2}, Ningyang Wang^{2

3}, Yaping Wang^{1

2}, Mengyao Liu^{2

3}, Qian Qing^{2

3}, Qihang Su^{2

3}, Yan Zou^{2

3}, Yang Liu^{4

2}

Affiliations

¹ School of Pharmacy, Henan University, Kaifeng 475004, China.
² Henan Key Laboratory of Brain Targeted Bio-Nanomedicine, School of Life Sciences & School of Pharmacy, Henan University, Kaifeng 475004, China.
³ Henan International Joint Laboratory of Nanobiomedicine, School of Life Sciences, Henan University, Kaifeng 475004, China.
⁴ Translational Medicine Center, Huaihe Hospital of Henan University, Henan University, Kaifeng 475004, China.

Abstract

Glioblastoma multiforme (GBM) is the most common type of malignant tumor of the central nervous system, characterized by aggressiveness, genetic instability, heterogenesis, and unpredictable clinical behavior. Disappointing results from the current clinical therapeutic methods have fueled a search for new therapeutic targets and treatment modalities. GBM is characterized by various genetic alterations, and RNA-based gene therapy has raised particular attention in GBM therapy. Here, we review the recent advances in engineered non-viral nanocarriers for RNA drug delivery to treat GBM. Therapeutic strategies concerning the brain-targeted delivery of various RNA drugs involving siRNA, microRNA, mRNA, ASO, and short-length RNA and the therapeutical mechanisms of these drugs to tackle the challenges of chemo-/radiotherapy resistance, recurrence, and incurable stem cell-like tumor cells of GBM are herein outlined. We also highlight the progress, prospects, and remaining challenges of non-viral nanocarriers-mediated RNA-based gene therapy.

Keywords: RNA therapy; gene therapy; glioblastoma; nanomedicine; non-viral nano-delivery.

Publication types

Review

Grants and funding

This research was funded by the National Natural Science Foundation of China (32101152), China Postdoctoral Science Foundation (2023M741014), the Youth Fund of the Henan Natural Science Foundation (242300420457), the Key Technologies R & D Program of Henan (232102230092), and Key Support Areas Project of the Innovation and Entrepreneurship Training Program for China University Students (202310475002).