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Gene Therapy for Human Malignant Brain Tumors

Rainov, Nikolai G., Ren, Huan·2003·The Cancer Journal
DOI 10.1097/00130404-200305000-00006
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Abstract

Purpose Brain tumors were the first human malignancy to be targeted by therapeutic transfer of nucleic acids into somatic cells, a process also known as gene therapy. Malignant brain tumor cells in the adult brain have some unique biologic features, such as high mitotic activity on an essentially postmitotic background and virtually no tumor spread outside of the central nervous system. Brain tumors seem therefore to offer major advantages in the design of tumor-selective gene therapy strategies, and the role of gene therapy in malignant glioma has been investigated since the late 1980s, initially in numerous laboratory studies and later on in clinical trials. Design Retrovirus has been one of the earliest recombinant virus vectors used in brain tumors. Experiments in cell culture and in animal models have demonstrated the feasibility of retrovirus-mediated transduction and subsequent killing of glioma cells by toxic transgenes. Phase I and II clinical studies in patients with recurrent malignant glioma have shown a favorable safety profile and some efficacy of retrovirus-mediated gene therapy. However, the only prospective, randomized, phase III clinical study of retrovirus gene therapy in primary malignant glioma failed to demonstrate significant extension of progression-free or overall survival. Adenovirus- and herpes simplex virus type 1-based vectors have been actively investigated along with retrovirus, but their clinical use is still limited, mostly because of safety c…

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Metadata

Authors
Rainov, N. G., & Ren, H.
Venue
The Cancer Journal
Publisher
Ovid Technologies (Wolters Kluwer Health)
Published
2003-05
Volume
9
Issue
3
Pages
180-188
ISSN
1528-9117
DOI
10.1097/00130404-200305000-00006
Cited by
48
References
0
Language
en

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CoChat. (2026). Citation check: Gene Therapy for Human Malignant Brain Tumors. https://app.cochat.ai/papers/10.1097/00130404-200305000-00006
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Rainov, N. G., & Ren, H. (2003). Gene Therapy for Human Malignant Brain Tumors. The Cancer Journal. https://doi.org/10.1097/00130404-200305000-00006