Cancer gene therapy using adeno-associated virus vectors
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Updated: Jul 10, 2026

High-Efficiency Transduction of Liver Cancer Cells by Recombinant Adeno-Associated Virus Serotype 3 Vectors
Published on: March 22, 2011
Keerang Park1, Wun-Jae Kim, Young-Hwa Cho
1Department of Biotechnology, Juseong Gene Therapy R&D Center, Juseong College, Chungbuk 363-794, Korea.
Abstract:
Gene therapy has offered highly possible promises for treatment of cancers, as many potential therapeutic genes involved in regulation of molecular processes may be introduced by gene transfer, which can arrest angiogenesis, tumor growth, invasion, metastasis, and/or can stimulate the immune response against tumors. Therefore, viral and non-viral gene delivery systems have been developed to establish an ideal delivery vector for cancer gene therapy over the past several years. Among the currently developed virus vectors, the adeno-associated virus (AAV) vector is considered as one of those that are closest to the ideal vector mainly for genetic diseases due to the following prominent features; the lack of pathogenicity and toxicity, ability to infect dividing and non-dividing cells of various tissue origins, a very low host immune response and long-term expression. Particularly, the most important attribute of AAV vectors is their safety profile in clinical trials ranging from CF to Parkinson's disease. Although adenovirus and several other oncolytic viruses have been more frequently used to develop cancer gene therapy, AAV also has many critical properties to be exploited for a cancer gene delivery vector. In this review, we will briefly summarize the basic biology of AAV and then mainly focus on recent progresses on AAV vector development and AAV-mediated therapeutic vectors for cancer gene therapy.
Insights
Adeno-associated virus (AAV) vectors show promise for cancer gene therapy due to their safety and efficacy. This review explores AAV vector development and applications in targeting tumors.
Area of Science:
- Oncology
- Gene Therapy
- Virology
Background:
- Gene therapy offers potential cancer treatments by introducing therapeutic genes to inhibit tumor growth and stimulate immune responses.
- Viral and non-viral gene delivery systems are crucial for effective cancer gene therapy.
- Adeno-associated virus (AAV) vectors are highly regarded for gene therapy due to their safety, broad infectivity, and low immunogenicity.
Purpose of the Study:
- To review the basic biology of adeno-associated virus (AAV).
- To focus on recent advancements in AAV vector development for cancer gene therapy.
- To highlight AAV-mediated therapeutic strategies against cancer.
Main Methods:
- Literature review of adeno-associated virus (AAV) biology and vector development.
- Analysis of AAV's properties relevant to cancer gene therapy.
- Summary of recent progress in AAV-mediated cancer treatment strategies.
Main Results:
- AAV vectors possess key features like non-pathogenicity, ability to infect various cell types, and low immune response, making them suitable for gene therapy.
- AAV vectors demonstrate a strong safety profile in clinical trials for various diseases.
- Despite frequent use of other oncolytic viruses, AAV holds significant potential for cancer gene delivery.
Conclusions:
- Adeno-associated virus (AAV) vectors are a promising platform for developing novel cancer gene therapies.
- Further research into AAV vector development can enhance its application in oncology.
- AAV-mediated gene therapy represents a viable strategy for cancer treatment with a favorable safety profile.
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