A review of chitosan in gene therapy: Developments and challenges

Dong, Liang; Li, Yanan; Cong, Hailin; Yu, Bing; Shen, Youqing

doi:10.1016/j.carbpol.2023.121562

Cited by 4 publications

(1 citation statement)

References 224 publications

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“…On the other hand, gene therapy has shown great promise in the treatment of cancer. , Small interfering RNA (siRNA) has been widely used in gene therapy. However, the naked gene can be easily degraded by nucleases in the circulation system, and the therapeutic effect of siRNA is largely dependent on the gene carrier. , Traditional gene carriers, including nanoparticles (NPs), peptides, and cationic polymers generally only function as carriers; they rarely have a therapeutic effect . Recently, Feng et al designed and synthesized serious cationic polythiophenes which can deliver DNA into tumor cells without additional polymer species.…”

Section: Introductionmentioning

confidence: 99%

Cationic Polythiophene as Gene Carrier and Sonosensitizer for Sonodynamic Synergic Gene Therapy of Hepatocellular Carcinoma

Chen,

Pang,

Peng

et al. 2024

ACS Biomater. Sci. Eng.

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Hepatocellular carcinoma (HCC) is one of the most lethal and highly malignant tumors. Sonodynamic therapy (SDT) is a new cancer treatment method. One of its unique advantages lies in the treatment of deep tumors due to its excellent tissue penetration ability caused by ultrasound (US). However, most sonosensitizers suffer from weak sonodynamic activity and poor tumor-targeting ability. In addition, small interfering RNA (siRNA) is a promising anticancer drug, and the efficacy of siRNA-based gene therapy largely depends on the cell impermeability of the gene carrier. Here, we designed and synthesized a cationic polythiophene derivative (PT2) that can be used as a siRNA carrier for gene therapy. Moreover, PT2 could generate singlet oxygen ( 1 O 2 ) and hydroxyl radicals (O 2•− ) under US irradiation, which suggests that PT2 could be used for SDT. Our study discovered that NUDT1 promoted HCC proliferation and inhibited intracellular ROS production. Therefore, si-NUDT1 was designed and synthesized. NUDT1 silencing can inhibit the proliferation of tumor cells and increase the production of intracellular ROS to further improve the efficacy of SDT. Then, si-NUDT1 assembled with PT2 and DSPE-PEG-FA to prepare a novel tumor-targeting nanodrug (PT2-siRNA@PEG-FA) for synergic SDT and gene therapy of HCC.

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