gene therapy

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Related to Human gene therapy: Somatic gene therapy

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.


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

gene therapy

[¦jēn ‚ther·ə·pē]
An experimental technique in which a normal gene is inserted into an organism to correct a genetic defect.
References in periodicals archive ?
Incorporating the TGase I promoter into vectors could be particularly useful for human gene therapy, vaccines, drug delivery, artificial skin production, and testing pharmaceuticals and cosmetics.
Related titles produced by Mary Ann Liebert include the journals Human Gene Therapy, DNA and Cell Biology, Tissue Engineering and Genetic Engineering News, a trade magazine for the biotechnology industry.
In addition, James Wilson, MD, director of the Philadelphia university's Institute for Human Gene Therapy, faces an FDA action that aims to bar him from ever conducting another human trial in the United States.
The case for liberating gene therapy from the current moral consensus is led by bioethicist LeRoy Walters and attorney Julie Gage Palmer in The Ethics of Human Gene Therapy, a provocative book that deserves wide and critical attention.
1936), working at the National Institutes of Health, conducted the first successful human gene therapy on a four-year-old girl who suffered from a genetic immune system disorder caused by the deficiency of a particular protein.
Only after those crucial points have been demonstrated in animals, he argued,should human gene therapy be attempted.
That's because Anderson's main goal is not simply to mark cells but to use foreign genes as a means of permanently curing genetic diseases, the application known as human gene therapy.
on process development and GMP manufacture of retroviral vector, adenoviral vector, adeno-associated viral vector and non-viral liposomal vector products for human gene therapy applications.
Human gene therapy company uniQure NV (NasdaqGS:QURE) revealed on Monday that it has received another USD53m from Bristol-Myers Squibb under the strategic collaboration to develop gene therapies to treat a range of cardiovascular diseases.
However the applicability of these vectors in human gene therapy depends on our ability to overcome the translational challenges between the mouse and human retinas.
The study was published in the journal Human Gene Therapy.
In a November 1999 paper in HUMAN GENE THERAPY, a team led by Robert L.