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, and any product 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 ?
Mochizuki et al., "Noninvasive in vivo delivery of transgene via adeno-associated virus into supporting cells of the neonatal mouse cochlea," Human Gene Therapy, vol.
"Human gene therapy" has been one of the most ambitious goals of biotechnology since the advent of molecular techniques for genetic modification in the 1970s.
Human gene therapy company uniQure NV (NasdaqGS:QURE) said on Friday that it has treated the first hemophilia B patient under the second high dose cohort of its ongoing AMT-060-01 Phase I/II trial.
The maturation of human gene therapy. Acta Paediatr 1996; 85:1261-1265.
The research was published by the journal Human Gene Therapy.
The study is described in an article published Instant Online in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc.
17 February 2012 - Dutch human gene therapy specialist Amsterdam Molecular Therapeutics BV (AMS:AMT) said on Friday it had agreed to be taken private by domestic uniQure BV in a move to secure funds for therapy development and repayment of debt.
Previously, he conducted one of the first human gene therapy studies at St.
Professor Salih Sanlioglu, chairman of the Human Gene Therapy Division of the Akdeniz University's Department of Medical Genetics, and his team's research rising hopes for patients with Type 1 diabetes made to the cover of October edition of the Human Gene Therapy magazine in the United States.
Three young adults who received gene therapy for a blinding eye condition remained healthy and maintained previous visual gains one year later, according to an August online report in Human Gene Therapy. One patient also noticed a visual improvement that helped her perform daily tasks, which scientists describe in an Aug.
UMass reported in a news release that the agreement gives Oxford BioMedica nonexclusive worldwide rights to use of the technology and certain exclusive rights to use the technology for human gene therapy involving lentiviral vectors.