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Rafael De la Garza-Ramos, Jessica V. Flores-Rodríguez, Juan Carlos Martínez-Gutiérrez, Alejandro Ruiz-Valls and Enrique Caro-Osorio

pathways have also been associated with these tumors. 17 , 42 , 64 , 84 The Principles Behind Genetics-Based Treatments for Cancer Gene therapy involves the artificial allocation of genetic material (complete genes or gene segments) into a host cell. General somatic gene therapy strategies include gene augmentation therapy, targeted killing of specific cells, and targeted inhibition of gene expression. 76 Gene augmentation therapy is useful for diseases caused by loss of function of a gene. Direct cell killing is possible when inserted genes cause cellular

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Mark M. Souweidane, Justin F. Fraser, Lisa M. Arkin, Dolan Sondhi, Neil R. Hackett, Stephen M. Kaminsky, Linda Heier, Barry E. Kosofsky, Stefan Worgall, Ronald G. Crystal and Michael G. Kaplitt

G ene therapy has long held the great potential to provide novel treatment options for neurological diseases. While the initial promise of this general field was tempered by slow progress, in recent years there has been a resurgence in the enthusiasm for gene therapy. This revival has been led by several new human clinical trials for a variety of neurodegenerative disorders including Parkinson, Alzheimer, and Canavan disease. 2 , 5 , 9 , 14 , 15 , 28 One of the unique limitations of CNS gene therapy is the blood-brain barrier, which prevents current

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Tord D. Alden, Debra D. Pittman, Elisa J. Beres, Gerald R. Hankins, David F. Kallmes, Benjamin M. Wisotsky, Kelvin M. Kerns and Gregory A. Helm

using gene therapy techniques to introduce the BMP gene into cells at the fusion site to achieve long-term, controllable BMP expression. The most successful technique for the introduction of therapeutic genes into cells in vivo is cellular transduction using viral vectors. 1, 8, 10–12, 20, 23 Although retroviruses, adenoassociated viruses, lentiviruses, and herpes viruses are all actively being investigated, adenoviruses are advantageous because of their high transduction rates and high production titers. However, adenoviruses can induce a strong immune

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Clemens M. F. Dirven, Jacques Grill, Martine L. M. Lamfers, Paul van der Valk, Angelique M. Leonhart, Victor W. van Beusechem, Hidde J. Haisma, Herbert M. Pinedo, David T. Curiel, W. Peter Vandertop and Winald R. Gerritsen

recurring meningiomas, despite incidental reports of tumor responses and stable disease after treatment with these modalities. 9, 12, 27, 34, 35, 43, 44, 46 Gene therapy, especially with adenoviral vectors, has developed as a promising option for treating tumors. Infection of a large number of tumor cells is crucial for the success of this type of therapy. Clinical trials in malignant gliomas have shown that the vectors used so far were unable to produce efficient transgene delivery into tumor cells, which explains the lack of significant therapeutic effect. 39, 40 Our

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Gregory A. Helm, Tord D. Alden, Elisa J. Beres, Sarah B. Hudson, Subinoy Das, Jonathan A. Engh, Debra D. Pittman, Kelvin M. Kerns and David F. Kallmes

inability to control growth factor release over time. In addition, the proteins cannot be given intravenously to induce bone deposition in specific tissues. The expression and secretion of BMPs through gene therapy techniques may overcome some of these limitations and improve the efficacy of BMP therapeutics. Several research groups have recently demonstrated that ex vivo gene therapy techniques may be useful for expressing BMP-2 in ectopic locations, leading to endochondral bone formation. 16, 25 Direct delivery of the BMP-2 gene via a first-generation adenoviral

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Jeffrey J. Laurent, K. Michael Webb, Elisa J. Beres, Kevin McGee, Jinzhong Li, Bert van Rietbergen and Gregory A. Helm

with the direct application of recombinant human BMP-2 on a variety of carrier matrices. 9, 34 Other groups have attempted to improve on these results by using gene therapy techniques to achieve more stable concentrations of BMPs at the fusion site. Both transfected stem cells and viral vectors have been used for this purpose. 16, 24, 26, 35–37 Several recent studies have shown that an adenoviral vector containing BMP-2 , -4 , -6 , -7 , and -9 genes formed ectopic bone when injected into the lower-extremity musculature of rats through processes similar to

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Mark H. Tuszynski, Hoi Sang U, John Alksne, Roy A. Bakay, Mary Margaret Pay, David Merrill and Leon J. Thal

The capacity to prevent neuronal degeneration and death during the course of progressive neurological disorders such as Alzheimer disease (AD) would represent a significant advance in therapy. Nervous system growth factors are families of naturally produced proteins that, in animal models, exhibit extensive potency in preventing neuronal death due to a variety of causes, reversing age-related atrophy of neurons, and ameliorating functional deficits. The main challenge in translating growth factor therapy to the clinic has been delivery of growth factors to the brain in sufficient concentrations to influence neuronal function. One means of achieving growth factor delivery to the central nervous system in a highly targeted, effective manner may be gene therapy. In this article the authors summarize the development and implementation of nerve growth factor gene delivery as a potential means of reducing cell loss in AD.

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Kyung-Ho Park, Jaeho Lee, Chul-Gyu Yoo, Young Whan Kim, Sung Koo Han, Young-Soo Shim, Seung-Ki Kim, Kyu-Chang Wang, Byung-Kyu Cho and Choon-Taek Lee

M alignant glioma is the most common type of primary brain tumor. The current standard treatment of malignant glioma is resection followed by radiation therapy with or without adjuvant chemotherapy. Despite this multidisciplinary approach, the prognosis of this disease is very poor. 16 Brain tumor may be an ideal target for gene therapy because it occurs in a closed space, is relatively small in size, is locally invasive in nature, and has a low metastatic potential. Many gene therapy strategies have been applied to brain tumor (for example, tumor suppressor

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Nobusada Shinoura, Lin Chen, Maqsood A. Wani, Young Gyu Kim, Jeffrey J. Larson, Ronald E. Warnick, Matthias Simon, Anil G. Menon, Wan Li Bi and Peter J. Stambrook

A n astrocytoma is a primary brain tumor that is amenable to retroviral gene therapy using a “suicide gene.” 4, 5 The neurons surrounding these tumors are in a nondividing, postmitotic state and the tumors grow locally, seldom metastasizing to another organ. One of the significant features of preclinical gene therapy experiments is the finding that experimental glioblastomas regressed completely after ganciclovir treatment, even when the tumors were comprised of a mixture of wild type— and herpes simplex virus—thymidine kinase (HSV-tk)—transduced cells. 24

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Masafumi Hiramatsu, Tomohito Hishikawa, Koji Tokunaga, Hiroyasu Kidoya, Shingo Nishihiro, Jun Haruma, Tomohisa Shimizu, Yuji Takasugi, Yukei Shinji, Kenji Sugiu, Nobuyuki Takakura and Isao Date

. 7 , 14 , 17 , 18 , 26 It has been reported that apelin plus VEGF effectively induced the formation of functional vessels that were larger than those induced with VEGF alone in a hindlimb ischemia model. 17 In this study, we evaluated whether combined gene therapy with VEGF plus apelin during indirect vasoreconstructive surgery enhances brain angiogenesis in a chronic cerebral hypoperfusion model in rats. Methods Animals and Surgical Procedures All animal procedures performed in this study were specifically approved by the Institutional Animal Care and Use