Tumors gene therapy

TSE (transmissible spongiform encephalopathies) 40 tubular reabsorption 155 tumor gene therapy 240 tumorigenicity 94 twinrix 134 tylosin 14 type I-cell 182... 

Rehemtulla A, Hall DE, Stegman LD, Prasad U, Chen G, Bhojani MS, Chenevert TL, Ross BD. Molecular imaging of gene expression and efficacy following adenoviral-mediated brain tumor gene therapy. Mol Imaging 2002 1 43-55. 

Combined chemotherapy/gene therapy of tumors based on activation of antitumor preparations with cytochromes P450 99MI38. 

Aboody KS, Najbauer J, Danks MK (2008) Stem and progenitor cell-mediated tumor selective gene therapy. Gene Ther 15 739-752... 

First clinical human gene therapy trials with polyplexes were performed using cancer vaccines based on autologous patient tumor cells. These were modified ex vivo with interleukin-2 pDNA. To obtain high level transfection rates of patient s primary tumor cells, Tf-PLL/pDNA polyplexes linked with inactivated endosomolytic adenovirus particles were applied [221]. Polymer-based in vivo human gene transfer studies were performed with PEGylated PLL polyplexes, delivering CFTR pDNA to the airway epithelium of cystic fibrosis patients [222],... 

To date, cellular and gene therapy products submitted to FDA have included clinical studies indicated for bone marrow marking, cancer, cystic fibrosis, AIDS, and inborn errors of metabolism and infectious diseases. Of the current active INDs approximately 78% have been sponsored by individual investigators or academic institutions and 22% have also been industry sponsored. In addition to the variety of clinical indications the cell types have also been varied. Examples include tumor infiltrating lymphocytes (TIL) and lymphocyte activated killer (LAK) cells, selected cells from bone marrow and peripheral blood lymphocytes, for example, stem cells, myoblasts, tumor cells and encapsulated cells (e.g., islet cells and adrenal chromaffin cells). 

Adenovirus A group of viruses that cause respiratory tract and eye infections. Adenoviruses used in gene therapy are altered to carry a specific tumor-fighting gene. [NIH]... 

Several gene therapies have received orphan drug designation these are treatments for cystic fibrosis, Gaucher disease, and metastatic brain tumor. 

Canote R, Du Y, Carling T, Tian F, Peng Z, Huang S (2002) The tumor suppressor gene RIZ in cancer gene therapy. Oncol Rep 9 57-60... 

Yoshida J, Mizuno M. Clinical gene therapy for brain tumors. Liposomal delivery of anticancer molecule to glioma. J Neurooncol 2003 65(3) 261-267. 

Currently, more than 400 human somatic cell gene therapy protocols are being tested. Most of these involve the use of genetically modified cells to treat noninherited diseases. For example, normal copies of the p53 tumor suppressor gene are inserted into lung tumors to halt tumor progression, and genetically modified cells have been used to create new coronary vessels in patients with coronary heart disease. Success has also been achieved in the treatment of hereditary disease (most notably, the recent successful treatment of X-linked severe combined immune deficiency see Clinical Correlate). 

Many diseases, such as hereditary metabolic defects and tumors, can still not be adequately treated. About 10 years ago, projects were therefore initiated that aimed to treat diseases of this type by transferring genes into the affected cells (gene therapy). The illustration combines conceivable and already implemented approaches to gene therapy for metabolic defects (left) and tumors (right). None of these procedures has yet become established in clinical practice. 

To reduce the side effects of cytostatic agents, new approaches are currently being developed on the basis of gene therapy (see p. 264). Attempts are being made, for example, to administer drugs in the form of precursors (known as prodrugs), which only become active in the tumor itself ( tumor targeting ). 

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