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Gene Therapy
Gene therapy is an approach to preventing and/or treating disease by replacing, removing or introducing genes or otherwise manipulating genetic material. Examples include adding a gene to a cell to produce a specific missing protein, using antisense molecules to prevent viral replication, and altering CD4 cells to make them resistant to HIV infection. Gene therapy may be introduced by direct injection or using a harmless viral vector to deliver genes into cells.
In most gene therapy studies, a "normal" gene is inserted into the genome to replace an "abnormal," disease-causing gene. To stimulate gene therapy, a carrier molecule called a vector must be used to deliver the therapeutic gene to the patient's target cells. Currently, the most common vector is a virus that has been genetically altered to carry normal human DNA. Viruses have evolved a way of encapsulating and delivering their genes to human cells in a pathogenic manner. Scientists have tried to take advantage of this capability and manipulate the virus genome to remove disease-causing genes and insert therapeutic genes.
Different types of viruses used as gene therapy vectors:
Retroviruses, Adenoviruses, Adeno-associated viruses, Herpes simplex viruses.
With gene therapy, target cells such as the patient's liver or lung cells are infected with the viral vector. The vector then unloads its genetic material containing the therapeutic human gene into the target cell. The generation of a functional protein product from the therapeutic gene restores the target cell to a normal state.
Gene Therapy on the Horizon:
Gene therapy researchers also are experimenting with introducing a 47th (artificial human) chromosome into target cells. This chromosome would exist autonomously alongside the standard 46 --not affecting their workings or causing any mutations. It would be a large vector capable of carrying substantial amounts of genetic code, and scientists anticipate that, because of its construction and autonomy, the body's immune systems would not attack it. A problem with this potential method is the difficulty in delivering such a large molecule to the nucleus of a target cell.
Learn more about gene therapy at the Comprehensive Cancer Center of Wake Forest University. As a national leader in cancer care & research and ranked among the best treatment facilities in the country, it is one of only 38 cancer centers in the nation designated by the National Cancer Institute (NCI) as a Comprehensive Cancer Center. NCI designation is an assurance of excellent care and comprehensive treatment options, including access to the latest clinical trials.
Gene Therapy
Copyright: Wake Forest University School of Medicine
and North Carolina Baptist Hospital. All rights reserved.
Medical Center Boulevard
Winston-Salem, NC 27157
(336) 716-2011 |
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