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Topic: Gene therapy

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	Gene Therapy
		Genes, which are carried on chromosomes, are the basic physical and functional units of heredity.
		Gene therapy is effectively used to treat two adult patients for a disease affecting nonlymphocytic white blood cells called myeloid cells.
		Gene therapy for treating children with X-SCID (sever combined immunodeficiency) or the "bubble boy" disease is stopped in France when the treatment causes leukemia in one of the patients.
	www.ornl.gov /sci/techresources/Human_Genome/medicine/genetherapy.shtml (2751 words)

	Gene Therapy for Cancer: Q & A - National Cancer Institute
		Gene therapy is an experimental treatment that involves introducing genetic material into a person’s cells to fight or prevent disease (see Question 2).
		A gene can be delivered to a cell using a carrier known as a “vector.” The most common types of vectors used in gene therapy are viruses (see Question 4).
		Gene therapy is being studied in clinical trials (research studies with people) for many different types of cancer and for other diseases.
	www.cancer.gov /cancertopics/factsheet/Therapy/gene (2277 words)

	Gene Therapy
		Human gene therapy is the administration of genetic material to modify or manipulate the expression of a gene or to alter the biological properties of living cells.
		Currently, gene therapy studies are examining a broad range of potential therapeutic interventions, including stimulating the body's immune reaction to tumors, inducing new blood vessels in the heart to alleviate heart attacks, and stopping the replication of HIV in AIDS patients.
		Gene therapy products present extraordinarily novel and controversial issues associated with cutting edge medical technology, ranging from the use of mouse and human viruses to produce gene vectors (carriers of genes), to the ethical and social issues involved with the potential for gene alteration in utero and other uses which could affect future generations.
	www.dhhs.gov /asl/testify/t000202b.html (3168 words)

	NHF \| Bleeding Disorders Information Center \| Gene Therapy: general information
		Hemophilia is considered an ideal disease for gene therapy because it's caused by a single malfunctioning gene, and only a small increase in clotting factor in the bloodstream could provide great medical benefits.
		If gene therapy raises levels of missing clotting factors by as little as 2% of normal, it is enough to prevent patients from suffering spontaneous hemorrhages into joints, brain and other organs, as happens with hemophilia.
		Although gene therapy could potentially replace treatment for hemophilia, it only corrects the defect in the hemophilic individual, not in their genetic line.
	www.hemophilia.org /bdi/bdi_gene.htm (446 words)

	Gene Therapy for Cancer
		Gene therapy is the insertion of a functional gene into the cells of a patient to correct an inborn error of metabolism, to alter or repair an acquired genetic abnormality, and to provide a new function to a cell.
		Gene therapy, because it is a modification of the human genome, generates a level of concern different from other therapies and therefore is deserving of continued public scrutiny.
		An emphasis for gene therapy for cancer should be put on improving the delivery of genes to target cells and on their "expression" and clinical trials using gene therapy for cancer should continue.
	www.cc.ndsu.nodak.edu /instruct/mcclean/plsc431/students/guojun.htm (4080 words)

	AMA (Professionalism) E-2.11 Gene Therapy
		Gene therapy involves the replacement or modification of a genetic variant to restore or enhance cellular function or to improve the reaction of non-genetic therapies.
		The fundamental difference between germ line therapy and somatic cell therapy is that germ line therapy affects the welfare of subsequent generations and may be associated with increased risk and the potential for unpredictable and irreversible results.
		Gene therapy should be pursued only after the availability or effectiveness of other possible therapies is found to be insufficient.
	www.ama-assn.org /ama/pub/category/8432.html (476 words)

	Gene Therapy
		For gene therapy, this is accomplished by changing the pattern of expression of the genes whose products may thus achieve the desired effect on the cellular phenotype.
		Gene therapy was initially conceptualized as a method to treat genetic diseases.
		In summary, it is not yet established that gene therapy for any indication results in improved health outcomes, nor is it established that the health outcomes from gene therapy are as good as or better than the health outcomes from existing therapies.
	www.regence.com /trgmedpol/medicine/med13.html (943 words)

	Gene Therapy - The ALS Association
		Gene therapy is the use of genetic instructions to produce a protein to treat a disorder or deficiency.
		Gene therapy in ALS may be able to target the glial cells as well as neurons, to produce positive effects.
		Gene therapy may be the way to provide a steady supply of trophic factors to neurons damaged in ALS, directly at the place where the damage exists.
	www.alsa.org /research/article.cfm?id=831 (1120 words)

	Gene therapy - Better Health Channel.
		At the present time, gene therapy is an experimental discipline and much research remains to be done before this approach to the treatment of disease will realise its full potential.
		A replaced, healthy gene would cure the individual, but not prevent their children from inheriting the original faulty gene, which is carried on the sperm and egg cells (called ‘germ’ cells).
		Gene therapy is an experimental form of treatment that works by replacing a faulty disease-causing gene with a healthy version.
	www.betterhealth.vic.gov.au /bhcv2/bhcarticles.nsf/pages/Gene_therapy (1273 words)

	FACSNET NewsBackgrounder \| Covering Gene Therapy
		When a scientist finds a genetic marker or the genes for a disease —; especially a common disease such as breast or colon cancer —; it is newsworthy mainly because of the therapeutic implications.
		Similarly, the much-publicized experiment on gene therapy for children who lack an immune system enzyme was a technical success, but its very high cost limits extending its use beyond experimentation.
		Hyperbole in the reporting of research is a special problem in the area of gene therapy because the public — and journalists — may be overly receptive to scientific findings that seem to promise solutions for devastating disease.
	www.facsnet.org /tools/nbgs/a_thru_h/c/covergene.php3 (1846 words)

	Gene therapy - Wikipedia, the free encyclopedia
		Gene therapy is the insertion of genes into an individual's cells and tissues to treat a disease, and hereditary diseases in particular.
		Gene C is the "normal" or "desirable" gene we want in the place of gene B. Thus, by re-engineering the virus so that gene B is replaced by gene C, while allowing gene A to properly function, this virus could introduce your 'good gene'- gene C into the host cell's genome without causing any disease.
		Gene therapy trials to treat severe combined immunodeficiency (SCID) were halted or restricted in the USA when leukemia was reported in three of eleven patients treated in the French Therapy X-linked SCID (XSCID) gene therapy trial.
	en.wikipedia.org /wiki/Gene_therapy (4538 words)

	Gene Therapy
		Inserting normal genes with correct information into the DNA of the cells that contain flawed genes, instead of repeatedly treating a patient with hemophilia with clotting factor, would allow those cells to make their own clotting factor.
		The therapy would be called somatic cell gene therapy and would not attempt to affect the germ line cells, which carry genetic information to the next generation.
		Other worries include the possibility that transferred genes could be "overexpressed," producing so much of the missing protein as to be harmful; that the viral vector could cause inflammation or an immune reaction, especially if administered repeatedly; and that the virus could be transmitted from the patient to other individuals or into the environment.
	www.personalmd.com /news/gene_therapy_030300.shtml (976 words)

	Gene Therapy
		The gene therapy team, led by Elizabeth Fenjves, Ph.D., is collaborating with Dr. Howard J. Federoff of the University of Rochester Medical Center, and is presently testing the Herpes Simplex Virus (HSV) system, which has been used in clinical human trials since 1998 and has an excellent safety profile.
		In collaboration with Dr. Andrew Stewart of the University of Pittsburgh and Dr. Norma Kenyon of the DRI, the gene therapy group has been able to safely and effectively transfect animal models with the HGF gene, and is in the process of adapting this technique in human islets.
		A major goal of the gene therapy team is to design a vector that can safely and effectively carry several genes into a cell in order to provide that cell with optimal protection.
	www.diabetesresearch.org /Research/EmergingTechnologies/GeneTherapy.htm (359 words)

	Gene Therapy - An Overview
		The types of gene therapy described thus far all have one factor in common: that is, that the tissues being treated are somatic (somatic cells include all the cells of the body, excluding sperm cells and egg cells).
		In contrast to this is the replacement of defective genes in the germline cells (which contribute to the genetic heritage of the offspring).
		Gene therapy in germline cells has the potential to affect not only the individual being treated, but also his or her children as well.
	www.accessexcellence.org /RC/AB/BA/Gene_Therapy_Overview.html (1284 words)

	Report and Recommendations of the Panel to Assess the NIH Investment in Research on Gene Therapy. This report concerns ...
		Gene therapy is a set of approaches to the treatment of human disease based on the transfer of genetic material (DNA) into an individual.
		Overzealous representation of clinical gene therapy has obscured the exploratory nature of the initial studies, colored the manner in which findings are portrayed to the scientific press and public, and led to the widely held, but mistaken, perception that clinical gene therapy is already highly successful.
		The Panel urges gene therapy investigators and their sponsors--be they academic, governmental, private, or industrial-to be more circumspect regarding the aims and accomplishments of clinical protocols when discussing their work with the scientific community, the public, and the media.
	www.nih.gov /news/panelrep.html (16879 words)

	Gene Therapy and Children
		Gene therapy carries the excitement of a cure-all for a host of diseases, the controversy surrounding the altering of human genes, and the promise of a type of medical treatment most of us would never imagine possible.
		Genes are composed of strands of a molecule called DNA and are located in single file within the chromosomes.
		Gene therapy's potential to revolutionize medicine in the future is exciting, and its expectations for curing and preventing childhood diseases is encouraging.
	kidshealth.org /parent/system/medical/gene_therapy.html (1305 words)

	Gene Therapy
		Thus, the rationale of the initial gene therapy study was that a murine fibroblast packaging cell line could be directly delivered into the region of brain tumor cells.
		Other gene therapy products are also being evaluated for their possible ability to turn off tumor cell growth once they are taken up by the tumor cell.
		Although this therapy is clearly in its infancy, it is the belief of those investigators exploring this technique that it should be utilized as soon as possible in children with malignant gliomas.
	www.childhoodbraintumor.org /gene_article.html (1954 words)

	Gene therapy
		Gene therapy is a rapidly growing field of medicine in which genes are introduced into the body to treat diseases.
		Gene therapy seeks to provide genes that correct or supplant the disease-controlling functions of cells that are not, in essence, doing their job.
		Gene therapy also is being considered as an approach to solving a problem associated with a surgical procedure known as balloon angioplasty.
	www.healthatoz.com /healthatoz/Atoz/ency/gene_therapy.jsp (4761 words)

	AMA (Genetics) Gene therapy
		Gene therapy is a novel approach to treat, cure, or ultimately prevent disease by changing the expression of a person’s genes.
		Gene therapy is in its infancy, and current therapies are primarily experimental, with most human clinical trials still in the research stages.
		In germline gene therapy, the patient’s egg or sperm cells are changed with the goal of passing on changes to their offspring.
	www.ama-assn.org /ama/pub/category/2827.html (844 words)

	Gene therapy : Cancerbackup (Site not responding. Last check: )
		Gene therapy is a very new type of treatment which is still being developed.
		Gene therapy means putting genetic material (DNA) into cells so that the cells can produce proteins which they do not usually produce.
		Gene therapy is a new technique that is being developed for the treatment of a number of different cancers.
	www.cancerbackup.org.uk /Treatments/Biologicaltherapies/Genetherapy (1143 words)

	Gene Therapy - J. Michael Mathis, Ph.D.
		Gene therapies delivered by adenovirus vectors have been quite successful in rodent models, and complete elimination of tumor cells is often achieved, even with poor transduction efficience in vivo.
		Secondly, once delivered the gene must be expressed by the transduced cells at a frequency and duration sufficient to affect viability of the cells.
		Thirdly, the gene therapy must be able to arrest tumor growth or reduce the tumor mass at the site of occurrence to be considered therapeutic.
	www.sh.lsuhsc.edu /gene_therapy/members/Mathis.htm (1780 words)

	Technology Review: Safer Gene Therapy
		In the therapy, a copy of a gene is delivered to a specific tissue to correct an abnormality -- for example, the lungs in cystic fibrosis.
		The gene is often delivered with viruses, which have evolved the ability to deliver their genes into human cells.
		Safer Gene Therapy by Guest (Joseph Ranghelli) 4/26/2006 12:00 AM The possibility of misuse exists in this terrorist threatened environment we live in.
	www.techreview.com /BioTech/wtr_16740,312,p1.html (659 words)

	Gene Therapy for Human Patients
		The treatment is called human gene therapy and is one of a series of emerging genetic techniques, commonly called genetic engineering, based on new knowledge about how genes work.
		The basic idea behind gene therapy is to insert normal genes with correct information into the DNA of the cells that contain malfunctioning genes.
		The best outcome of human gene therapy would be a single treatment that would correct enough cells to provide a permanent cure for the patient's disease.
	www4.od.nih.gov /oba/rac/cover.htm (2462 words)

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