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, 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 ?
"The recently released papers by the Royal Society Te Aparangi note there are considerable benefits that gene editing can bring to our lives, particularly in health," Environment Minister David Parker said.
These forward-looking statements include, without limitation, statements regarding the Company's ability to develop and commercialize product candidates to address genetic diseases with the Company's proprietary technologies; the anticipated benefit from investingin a diverse pipeline of products; Sangamo's ability to enhance efficacy of in vivo gene editing, increase potency with Sangamo's updated gene editing reagents and integrate such improvements into its gene editing clinical development programs, as well as the timing of commencement of such programs and the anticipated benefits therefrom and Sangamo's 2019 financial guidance related to cash, cash equivalents, and investments, anticipated operating expenses and cash runway.
Gene editing, or genome engineering, is a type of genetic engineering in which DNA is inserted, deleted, modified or replaced in the genome of a living organism.
Further, the pivotal technologies prevalently used to propel gene editing are cluster regularly interspaced short palindromic repeats (CRISPR) associated protein 9 (Cas9), zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), namely.
CRISPR has captivated scientists because it's a very simple way to do gene editing, although it's so new that its risks are not fully known.
In the Fourth Industrial Revolution, new gene editing tools are likely to overtake biological evolution
But Jaydee Hanson of the Center for Food Safety said gene editing could also be used to make more significant changes, including those that would never happen in nature, and said oversight is necessary.
(NASDAQ: TECH), a holding company for biotechnology and clinical diagnostic brands, has acquired all of the stock of Minneapolis-based gene editing firm B-MoGen Biotechnologies Inc., the company said.
In the further work, the team hopes to use the gene editing technology, known as CRISPR, to remove a section of the birds' DNA responsible for producing a protein called ANP32, on which all flu viruses depend to infect a host.