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Treatment of Diseases by Gene Therapy

Another form of gene therapy to treat cancer involves oncolytic virotherapy. This involves the use of oncolytic vectors, which are virus-designed to home and kill the tumor cells without harming the normal cells in the body. The cancer cells are killed by cell lysis as a result of the production of cytotoxic proteins or due to the propagation of the virus itself. The viruses that have been used to produce oncolytic vectors include adenovirus, vaccinia, reovirus, HSV-1 and Newcastle disease virus. [Pg.239]

The ongoing clinical trials include the use of adenovirus and herpes virus vectors. One example of adenoviral vector is ONYX-015, which lacks E1B protein, required for replication with a normal p53 pathway and RNA export during viral replication. It has been used to treat squamous cell carcinoma of the head and neck and has also been tested as a preventive treatment for oral precancerous tissue. The concept behind using this vector is that ONYX-015 will proliferate in p53 pathway-deficient tumor cells and kill them. [Pg.239]


Techniques of molecular biology are used in the prevention and treatment of disease. For example, recombinant DNA techniques provide human insulin for the treatment of diabetes. Factor VIII for the treatment of hemophilia, and vaccines for the prevention of hepatitis. Although treatment of disease by gene therapy is in the experimental phase of development, the possibilities are limited only by the human imagination and, of course, by ethical considerations. [Pg.297]

Human gene therapy Treatment of disease by insertion of new genetic material or permanent modification of existing genes. [Pg.320]

Finally, in 2000, successful treatment of an X-linked form of SCID (SCID-Xl) by gene therapy was reported by a French medical team. Almost a year after a normal gene was introduced into their cells, two children were still synthesizing the enzyme that they lacked. This was the first success for gene therapy in curing a disease after years of effort. [Pg.537]


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