gene therapy

(redirected from Antibody genes)
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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 ?
They had an adverse expression profile involving high inflammation and low antiviral and antibody gene expression.
It also identifies 11 possible large DNA insertions and deletions of antibody-encoding genes in the region that determine our antibody gene count/diversity and, in some cases, have been implicated in disease susceptibility.
Was there something special about the antibody genes that allowed for production of such a large potential pool of different specificities to be manufactured?
Antibody genes have evolved to be naturally templated to attract AID to the binding region.
When the body is fending off an infection, B cells can mutate their antibody genes randomly until they produce one that 'sticks' to the invading antigen.
Because the antibody genes function continuously in most white blood cells, says Hengartner, the swap keeps ced-9 permanently turned on, letting some old white cells outlive their usefulness and proliferate as cancer.
BioInvent's proprietary antibody discovery platform, n-CoDeR([R]), contains over 20 billion (2 x1010) human antibody genes, a far greater number than those naturally available in the human immune system.
The team of researchers identified white blood cells from the patients that made antibodies against flu virus, and then isolated the antibody genes from individual cells.
The method covered by the new patent enables this by preventing the expression of mouse antibody genes, so that they can be functionally replaced with human antibody genes, while leaving intact the rest of the mouse immune system.
Although the Whitehead researchers do not yet know the exact function of their new find -- which they have named RAG-1, for recombination activating gene -- they suspect it might encode a recombinase that reshuffles antibody genes.
Using bioengineering techniques, the antibody genes were cloned and the resulting antibodies were tested for their ability to detect BoNT in a selection of drinks, including milk.
Then they develop a library of the antibody genes in bacterial cells, each gene coding for just one of the antibodies elicited by an antigen.