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Mechanism Of Transfer In Gene Therapy

2068 words - 8 pages

Mechanism of Transfer in Gene Therapy
Abstract:
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 gene therapy experiments so far have been in vitro – in controlled environments, which are test tubes in this case. This is so because gene therapy is a new field and has the potential to complicate patients’ conditions even more, and there are ethical concerns.
Somatic and germline gene therapy are the two types of gene therapy. Somatic cells are body cells whereas germline cells are sex cells or embryos. Alterations to germline cells allow the changes in defective or undesired cells to be passed on to future generations. This is especially useful for families with histories of inherited diseases. Germline cells include sperm cells, egg cells, and zygotes. If the changes are made before the embryo begins to develop or develops much, then the genes may be integrated into the DNA and passed on to progeny. The sperm and egg are easy to manipulate, especially the egg because it is a large cell, but there is only a possibility that the inserted gene will be integrated. If gene therapy is done on a zygote, it must be done in vitro, and then the cells must be inserted into a surrogate mother. Despite its advantages and potential for improved gene therapy in the future, germline gene therapy is a much-debated field.
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