Gene therapy is the transfer of “normal” genes into the body to replace defective or undesired genes. The transfer may be in somatic or germline cells and may take place in vivo or in vitro. The DNA may be inserted in a retrovirus, adenovirus, adeno-associated virus, herpes simplex virus, or liposome, or it may be naked DNA. The vector travels to a target cell and inserts the gene, which goes to the host cell’s nucleus and may integrate into the genome. In cells with dominant negative genes, resulting protein must be prevented from translation or transcription using oligonucleotides or antisense technology. Though many obstacles delay the development of gene therapy, this new field will surely improve the future of medicine.
Mechanism of Transfer in Gene Therapy
Gene therapy is a method of replacing defective or undesired genes in the body with “normal” genes. A vector is re-engineered to deliver the gene to a target cell. Then the gene is transferred to the cell’s nucleus and must be activated in order to function. The gene must be integrated into the cell’s genome in order for it to continue to function and be replicated. Side effects are possible because the vector may be detected as a foreign substance by the body’s immune system. Gene therapy may take place either in vivo or in vitro, the transfer may be in either somatic or germline cells, and a variety of viral and non-viral vectors may be used.
In vivo gene therapy is done inside a living organism. This is accomplished by inhalation, oral administration, intramuscular injection, or intravenous administration (Brooks, 24). Currently, most in vivo experiments have taken place in animals other than humans. Most gen...
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...ocated, ensure that gene therapy will be a revolutionary aspect of medicine’s future.
Brooks, Gavin. Gene Therapy: The use of DNA as a drug. London: Pharmaceutical Press, 2002.
“Gene Therapy.” Human Genome Project Information. 18 November 2005. U.S. Department of Energy and the National Institutes of Health. 28 July 2007.
“Gene Therapy: Molecular Bandage?” Learn.Genetics. 2007. The University of Utah, Genetic Science Learning Center. 28 July 2007.
Januario E. Castro, Thomas J. Kipps. “Chapter 26. Principles of Gene Transfer for Therapy.” Williams Hematology, 7th Edition. United States of America: The McGraw-Hill Companies, Inc., 2006. 28 July, 2007.
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