gene therapy

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gene therapy,

the use of genesgene,
the structural unit of inheritance in living organisms. A gene is, in essence, a segment of DNA that has a particular purpose, i.e., that codes for (contains the chemical information necessary for the creation of) a specific enzyme or other protein.
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 and the techniques of genetic engineeringgenetic engineering,
the use of various methods to manipulate the DNA (genetic material) of cells to change hereditary traits or produce biological products. The techniques include the use of hybridomas (hybrids of rapidly multiplying cancer cells and of cells that make a
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 in the treatment of a genetic disorder or chronic disease. There are many techniques of gene therapy, all of them still in experimental stages. The two basic methods are called in vivo and ex vivo gene therapy. The in vivo method inserts genetically altered genes directly into the patient; the ex vivo method removes tissue from the patient, extracts the cells in question, and genetically alters them before returning them to the patient.

The challenge of gene therapy lies in development of a means to deliver the genetic material into the nuclei of the appropriate cells, so that it will be reproduced in the normal course of cell division and have a lasting effect. One technique involves removing cells from a patient, fortifying them with healthy copies of the defective gene, and reinjecting them into the patient. Another involves inserting a gene into an inactivated or nonvirulent virus and using the virus's infective capabilities to carry the desired gene into the patient's cells. A liposomeliposome
, microscopic, fluid-filled pouch whose walls are made of layers of phospholipids identical to the phospholipids that make up cell membranes. Liposomes are used to deliver certain vaccines, enzymes, or drugs (e.g., insulin and some cancer drugs) to the body.
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, a tiny fat-encased pouch that can traverse cell membranes, is also sometimes used to transport a gene into a body cell. Another approach employing liposomes, called chimeraplasty, involves the insertion of manufactured nucleic acid molecules (chimeraplasts) instead of entire genes to correct disease-causing gene mutations. Once inserted, the gene may produce an essential chemical that the patient's body cannot, remove or render harmless a substance or gene causing disease, or expose certain cells, especially cancerous cells, to attack by conventional drugs.

Gene therapy was first used in humans in 1990 to treat a child with adenosine deaminase deficiency, a rare hereditary immune disorder (see immunityimmunity,
ability of an organism to resist disease by identifying and destroying foreign substances or organisms. Although all animals have some immune capabilities, little is known about nonmammalian immunity.
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). Gene therapy has since been used experimentally to treat a number of conditions, including advanced metastatic melanoma, a myeloid disorder, and a rare hereditary condition that leads to severely impaired vision. Despite the hope that gene therapy can be used to treat cancer, genetic diseases, and AIDS, there are concerns that the immune system may attack cells treated by gene therapy, that the viral vectors could mutate and become virulent, or that altered genes might be passed to succeeding generations. In a few instances trials have been halted when a patient has died or developed disease after undergoing gene therapy.

In the United States, gene therapy techniques must be approved by the federal government. The Recombinant DNA Advisory Committee of the National Institutes of Health oversees gene therapy experiments. Like drugs, products must pass the requirements of the Food and Drug AdministrationFood and Drug Administration
(FDA), agency of the Public Health Service division of the U.S. Department of Health and Human Services. It is charged with protecting public health by ensuring that foods are safe and pure, cosmetics and other chemical substances harmless, and
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. Gene therapy is a competitive and potentially lucrative field, and patents have been awarded for certain techniques.

Bibliography

See J. Lyon and P. Gorner, Altered Fates: Gene Therapy and the Retooling of Human Life (1995).

gene therapy

[¦jēn ‚ther·ə·pē]
(genetics)
An experimental technique in which a normal gene is inserted into an organism to correct a genetic defect.
References in periodicals archive ?
The bovine production system is created by introducing the full, unrearranged sequences for both the human heavy and light chain antibody genes into cows, using a human artificial chromosome (HAC) vector, developed by Hematech's collaborators at the Kirin Brewery Company.
This summer, by sequencing tens of millions of antibody genes per person - more than 100 times more genes than can be sequenced with existing technology - a research team led by Harlan Robins, Ph.
In the human genome, rearrangement of antibody genes can enable the immune system to target infection more effectively.
Vybion is developing a humanized version of the monoclonal antibody genes, developing the expression lines and the process for manufacturing the product.
We believe, that the success of our collaboration with Hematech in producing transchromosomic calves carrying human antibody genes complements and extends our previous work in mice on human monoclonal antibodies, and opens up a vast new field of potential antibody therapeutic applications," said Isao Ishida, Ph.
The antibody genes are genetically engineered into the phage DNA (genotype) and introduced into bacterial host cells, which secrete phage particles on their cell surfaces.
Individual B cells selected using the technology are isolated and the antibody genes can be directly introduced into a manufacturing cell line.
The collaboration is based on GenPharm's HuMAb-Mouse(TM) strain, which contains functional human antibody genes.
Medarex's HuMAb-Mice are transgenic, meaning that the mouse genes for creating antibodies have been inactivated and replaced by human antibody genes.
However, we have seen changes over the past few years with several antibody-based products reaching the market; MedImmune's (Nasdaq: MEDI) Synagis, Centocor's Remicade, Genentech's (NYSE: DNA) Herceptin, and Protein Design Lab's (Nasdaq: PDLI) Zenapax, all of them are partially or fully humanized, meaning that human antibody genes are incorporated into producing these antibodies.
These unrearranged human antibody genes rearrange to form fully human antibodies in the transgenic mice.