<I>In vitro</I> Efficacy of mda-7 Gene for Hepatocellular Carcinoma Gene Therapy Mediated by Human Ribosomal DNA Targeting Vector*

Hepatocellular carcinoma (HCC) is the most common form of primary liver cancer and the fifth most common cancer worldwide. HCC is recognized as the fourth most common cause of cancer related deaths worldwide due to the lack of effective early diagnostic tools, which often leads to individuals going undiagnosed until the cancer has reached late stage development. The current FDA approved treatments for late stage HCC provide a minimal increase in patient survival and lack tumor specificity, resulting in toxic systemic side effects. Gene therapy techniques, such as chimeric antigen receptor (CAR)-T Cells, viral vectors, and nanoparticles, are being explored as novel treatment options in various genetic diseases. Pre-clinical studies using gene therapy to treat in vitro and in vivo models of HCC have demonstrated potential efficacy for use in human patients. This review highlights genetic targets, techniques, and current clinical trials in HCC utilizing gene therapy.

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Water-soluble Schiff base Cu(II) and Zn(II) complexes: Synthesis, DNA targeting ability and chemotherapeutic potential of Cu(II) complex for hepatocellular carcinoma - in vitro and in vivo approach

Applied Organometallic Chemistry ◽

10.1002/aoc.3739 ◽

2017 ◽

Vol 31 (10) ◽

pp. e3739 ◽

Cited By ~ 13

Author(s):

Narayanaperumal Pravin ◽

Ganesan Kumaravel ◽

Raju Senthilkumar ◽

Natarajan Raman

Keyword(s):

Hepatocellular Carcinoma ◽

Schiff Base ◽

Water Soluble ◽

Dna Targeting ◽

Targeting Ability

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Aptamer-mediated gene therapy enhanced antitumor activity against human hepatocellular carcinoma in vitro and in vivo

Journal of Controlled Release ◽

10.1016/j.jconrel.2017.05.017 ◽

2017 ◽

Vol 258 ◽

pp. 130-145 ◽

Cited By ~ 13

Author(s):

Shuangli Xiao ◽

Zhongbing Liu ◽

Ruolan Deng ◽

Chunhong Li ◽

Shaozhi Fu ◽

...

Keyword(s):

Hepatocellular Carcinoma ◽

Gene Therapy ◽

Antitumor Activity ◽

Human Hepatocellular Carcinoma

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Investigation of hrDNA targeting vector-mediated tumor-specific suicide gene therapy for hepatocellular carcinoma

Chinese Science Bulletin ◽

10.1007/s11434-006-2120-2 ◽

2006 ◽

Vol 51 (19) ◽

pp. 2342-2350 ◽

Cited By ~ 1

Author(s):

Lina Wang ◽

Zhigang Xue ◽

Zhuo Li ◽

Jinfeng Xue ◽

Xionghao Liu ◽

...

Keyword(s):

Hepatocellular Carcinoma ◽

Gene Therapy ◽

Suicide Gene ◽

Suicide Gene Therapy ◽

Targeting Vector

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Effect of Diphtheria Toxin-Based Gene Therapy for Hepatocellular Carcinoma

Cancers ◽

10.3390/cancers12020472 ◽

2020 ◽

Vol 12 (2) ◽

pp. 472 ◽

Cited By ~ 1

Author(s):

Kenya Kamimura ◽

Takeshi Yokoo ◽

Hiroyuki Abe ◽

Norihiro Sakai ◽

Takuro Nagoya ◽

...

Keyword(s):

Hepatocellular Carcinoma ◽

Gene Therapy ◽

Cell Growth ◽

Gene Delivery ◽

Diphtheria Toxin ◽

Mice Model ◽

Novel Therapy ◽

Liver Cancers ◽

Afp Promoter

Hepatocellular carcinoma (HCC) is a major global malignancy, responsible for >90% of primary liver cancers. Currently available therapeutic options have poor performances due to the highly heterogeneous nature of the tumor cells; recurrence is highly probable, and some patients develop resistances to the therapies. Accordingly, the development of a novel therapy is essential. We assessed gene therapy for HCC using a diphtheria toxin fragment A (DTA) gene-expressing plasmid, utilizing a non-viral hydrodynamics-based procedure. The antitumor effect of DTA expression in HCC cell lines (and alpha-fetoprotein (AFP) promoter selectivity) is assessed in vitro by examining HCC cell growth. Moreover, the effect and safety of the AFP promoter-selective DTA expression was examined in vivo using an HCC mice model established by the hydrodynamic gene delivery of the yes-associated protein (YAP)-expressing plasmid. The protein synthesis in DTA transfected cells is inhibited by the disappearance of tdTomato and GFP expression co-transfected upon the delivery of the DTA plasmid; the HCC cell growth is inhibited by the expression of DTA in HCC cells in an AFP promoter-selective manner. A significant inhibition of HCC occurrence and the suppression of the tumor marker of AFP and des-gamma-carboxy prothrombin can be seen in mice groups treated with hydrodynamic gene delivery of DTA, both 0 and 2 months after the YAP gene delivery. These results suggest that DTA gene therapy is effective for HCC.

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