21. Genome.gov | Talking Glossary: "gene Therapy" Home Educational Resources Talking Glossary. Back to the glossary. Pronounciation of gene therapy Dr. Michael Blaese defines gene therapy. related http://www.genome.gov/glossary.cfm?key=gene therapy

22. NHF | Bleeding Disorders Information Center | Gene Therapy: General Information gene therapy might eventually help people with hemophilia begin The future for gene therapy in hemophilia is continuing albeit at a moderate pace. http://www.hemophilia.org/bdi/bdi_gene.htm

This section of our Web site sponsored by: Gene Therapy Pages: gene therapy (main) gene therapy FAQ gene therapy articles ( HemAware gene therapy trials Gene Therapy Gene therapy represents a new field of medicine, one which can potentially cure many genetic diseases such as bleeding disorders. Patients with inherited diseases such as hemophilia usually lack a functional protein, which can be replaced with periodic protein replacement therapy. An alternative is to add a normal gene to substitute for the defective one. The normal gene, if delivered to the right cells and in sufficient quantity, can continuously make the normal version of the defective protein, restoring normal clotting mechanisms. However, this is a new and highly experimental field. Many questions remain unanswered and will only be answered through a series of clinical studies, some of which are ongoing using the genes for factors VIII and IX. There have been many failures and disapointments when investigators have found that the technology for delivering the genes was inadequate when used in humans. Sometimes investigators have underestimated the complexity of the diseases they were attempting to cure. Because of some recent successes, hemophilia has emerged as the top candidate for a genetic disease most likely to benefit from gene therapy.

23. Hardin MD : Gene Therapy From the University of Iowa s Hardin MD, Internet sources on gene therapy. http://www.lib.uiowa.edu/hardin/md/genetherapy.html

Gene therapy Return to Hardin MD : Home Medical Pictures People interested in this subject often clicked these ... Genetic Disorders Google Directory : Gene therapy MEDLINE plus National Library of Medicine Yahoo Directory : Science : Biology : Genetics : ... : Gene therapy Gene therapy US Government Hardin Library for the Health Sciences , University of Iowa Please send comments to hardin-webmaster@uiowa.edu The URL for this page is http://www.lib.uiowa.edu/hardin/md/genetherapy.html Last updated Tuesday, Sep 12, 2006 [human gene therapy, genes][25531

24. Fundamentals Of Gene Therapy A broken gene eliminates the production of an enzyme essential for the development of a normal immune system. Scientists isolated the normal copy of the http://www.fda.gov/fdac/features/2000/gene.html

Fundamentals of Gene Therapy Recently, French researchers reported dramatic results in treating a disease called severe combined immune deficiency (SCID), the disorder suffered by David, The Boy in the Bubble. A broken gene eliminates the production of an enzyme essential for the development of a normal immune system. Scientists isolated the normal copy of the gene and packaged it into a vector. In the laboratory, they then used the vector to transport the gene into the patient's own bone marrow cells. Bone marrow cells create the immune system. The treated bone marrow cells are then given back to the patient in a germ-free isolation room, where they reconstitute a normal, functioning immune system, freeing the patient from the need to remain in isolation. Table of Contents Return to Main Article FDA Home Page zwr 2000-AUG-14

25. Gene Therapy Systems Commercial firm providing reagents for gene delivery and gene therapy research, including unique pGeneGrip fluorescently labeled plasmid vectors and http://www.genetherapysystems.com/

26. Department Of Health: Gene Therapy Advisory Committee The gene therapy Advisory Committee (GTAC)is the UK national research ethics committee for gene therapy clinical research according to the Medicines for http://www.advisorybodies.doh.gov.uk/genetics/gtac/

DH Home You are here: Advisory bodies GTAC About GTAC What's new 'Flagging' project - monitoring patients Applying to GTAC to conduct clinical trials ... Contact GTAC Gene Therapy Advisory Committee (GTAC) Latest information: Important information for applicants considering applying to GTAC from 2008 Integrated Research Application System (IRAS) information Integrated Research Application System (IRAS) information in Rich Text Format GTAC is the UK national research ethics committee (REC) for gene therapy clinical research according to the Medicines for Human Use (Clinical Trials) Regulations 2004 ( http://www.opsi.gov.uk/si/si2004/20041031.htm , see article 14(5)). It is the only UK ethics committee empowered to approve clinical trials of gene therapy products according to the definition given in Part IV of Directive 2003/63/EC (amending Directive 2001/83/EC): "… [a] gene therapy medicinal product shall mean a product obtained through a set of manufacturing processes aimed at the transfer, to be performed either in vivo or ex vivo, of a prophylactic, diagnostic or therapeutic gene (i.e. a piece of nucleic acid), to human/animal cells and its subsequent expression in vivo. The gene transfer involves an expression system contained in a delivery system known as a vector, which can be of viral, as well as non-viral origin. The vector can also be included in a human or animal cell."

27. Gene-Therapy Deaths Raise Ethics Issues After nearly two decades of research, success is not a word generally associated with gene therapy. It s even led to deaths that some experts believe should http://www.wired.com/medtech/genetics/news/2007/08/gene_therapy

@import "/css/toolbox_article_bottom.css"; @import "/css/article.css"; @import "/css/google_ad.css"; @import "/css/wiredcomment.css"; Subscribe Renew Give a Gift Change Address ... WIRED Science Top Stories Magazine Wired Blogs All Wired Med-Tech Genetics Gene Therapy: Is Death an Acceptable Risk? By Brandon Keim Jolee Mohr, 36, died during a clinical trial using gene therapy to treat rheumatoid arthritis. The photograph was taken on July 1, the day before she received the injection. Photo: Courtesy of the Mohr family A 36-year-old woman with rheumatoid arthritis died in July, while participating in a gene-therapy clinical trial. Some experts say she shouldn't have received such an unpredictable, potentially dangerous treatment in the first place. Jolee Mohr was married, the mother of a 5-year-old daughter, and worked at the Secretary of State's office in her hometown of Springfield, Illinois. By all accounts she was able to lead a full and active life, with existing drugs keeping her disease under control. The Food and Drug Administration and the National Institutes of Health are still studying whether the trial therapy played a role in Mohr's death . But a sudden infection raged through her body and caused her organs to fail just after the experimental treatment was injected into her right knee, which has raised suspicion that her death was linked to the therapy.

28. BSP :: Current Gene Therapy Home Page Contains expert indepth reviews on all aspects of gene therapy. http://www.bentham.org/cgt/index.htm

Home News - Browse Titles - New titles ... - Free E-Updates Access Bentham Online: - Purchase Articles Online - BenthamDirect - Catalogs - Free Online ... Conferences Marketing Opportunities: - Advertise with us Partners - Marketing Exchanges Authors: - Manuscript Submission - Order Reprints and Single Issues - Permission ... Contact Us Current Gene Therapy ISSN: 1566-5232 - Volume 8, 6 Issues, 2008 Current Gene Therapy is an interdisciplinary journal focused on providing the readership with current and comprehensive reviews on all aspects of gene therapy. The journal is aimed at all academic and industrial scientists who are interested in understanding either the pre-clinical or clinical research on gene therapy. The journal serves as an expert forum to convey all the latest research on gene transfer and gene expression analysis, animal models, vector development and human clinical applications for the treatment of disease. First impact factor (2006): 3.68

29. Gene Therapy And Genomics gene therapy Clinical Trials find out what countries trials are underways in, what diseases are being addresses by therapy, and more. http://www.brightsurf.com/l_gene_therapy.html

[ Home ] Earth Science Alternative Energy Anthropology and Archaeology Earthquakes and Volcanoes Environment and Nature News ... Tsunami Space Science Astronomy and Space News Black Holes Chandra X-Ray Observatory Extrasolar Planets ... Space Weather Life Science Animal News Biotechnology and Genetics Brain Research Human Cloning ... Whales and Whaling Gene Therapy and Genomics Gene Therapy Clinical Trials - find out what countries trials are underways in, what diseases are being addresses by therapy, and more. From the Journal of Gene Medicine. What is Gene Therapy? - overview from Jim Wilson of the University of Pennsylvania's Institute for Human Gene Therapy . Introduction Jim Wilson What is Gene Gene therapy offers hope for anaemia sufferers Genetic Testing and Gene Therapy: What They Mean To You and Your Family - questions and answers from Ask NOAH. In the Service of Science - report on alleged regulatory and monitoring shortcomings over gene therapy trials in the U.S. and Canada. From CBC News' The Magazine. Introduction to Gene Therapy - primer on the subject, originally designed to supplement a course taught at Vanderbilt University.

30. Online NewsHour: Gene Therapy -- February 2, 2000 As gene therapy trials come under fire for underreporting harmful side effects, Congress begins investigating the proper balance between regulation and http://www.pbs.org/newshour/bb/health/jan-june00/gene_therapy_2-2.html

var gaJsHost = (("https:" == document.location.protocol) ? "https://ssl." : "http://www."); document.write(unescape("%3Cscript src='" + gaJsHost + "google-analytics.com/ga.js' type='text/javascript'%3E%3C/script%3E")); GENE THERAPY February 2, 2000 As gene therapy trials come under fire for underreporting harmful side effects, Congress begins investigating the proper balance between regulation and research. Susan Dentzer reports on developments in controversial gene therapy treatments. The Health Unit is a partnership with the Henry J. Kaiser Family Foundation. Health Spotlight on Gene Therapy Dec. 8, 1999: Controversial Therapy Dec. 2, 1999: Breaking the Code Nov. 30, 1999: Medical Errors Focus: Internet Rx Browse the NewsHour's coverage of health. American Society of Gene Therapy Dr. W. French Anderson SUSAN DENTZER: The promise of gene therapy has long seemed spectacular: Prevent or cure illness by injecting healthy copies of defective or missing genes into the body. But the death of a teenager last fall in a controversial gene therapy clinical trial focused a harsh spotlight on the ways these experiments are being carried out and monitored. That was the subject of a senate subcommittee hearing today chaired by Republican Senator William Frist of Tennessee.

31. University Of North Carolina At Chapel Hill's Gene Therapy Department Working to translate gene therapy research from the laboratory bench into Phase I clinical trials for the treatment of hematologic and endocrine diseases. http://genetherapy.unc.edu/

Research Samulski Lab Kafri Lab Monahan Lab ... Click here to learn more about our gene therapy research. Announcements March 2006 The first clinical trial for Duchenne Muscular Dystrophy begins, utilizing AAV vectors developed and produced in Dr. Samulski's laboratory. Click here for more information July 2005 The Gene Therapy Center would thank Dr. Mark Atkinson for speaking at the First Annual Diabetes Research Seminar to Memorialize Jerri Coleman. July 2004 In memory of Jerri Coleman, the Gene Therapy Center has established a memorial fund for diabetes research in her name. Click here for more information. March 2004 - Drs. Stilwell and Samulski published a breakthrough study on the safety of AAV virion shells. They found that viral capsids may influence gene expression independent from their packaged DNA. AAV and adenovirus were compared using DNA microarrays for their effects on cellular gene expression. AAV was found to have minimal impact on gene expression in the cell, while adenovirus activated the expression of genes associated with toxicity and the immune response. These findings indicate that AAV may be a safer and more effective form of transduction. Click here for more information about this study.

32. ACGT® - Alliance For Cancer Gene Therapy - National Grants For Cancer Research The alliance for Cancer gene therapy is the only public charity in the nation dedicated exclusively to investing in research into cancer gene therapies. http://www.acgtfoundation.org/

About ACGT Founder's Message Why ACGT is Unique National Reach ... Contact ACGT Learn more about our mission, our vision for cancer gene therapy research, our governance, and ACGT alliances across the country. ABOUT ACGT Learn the definition and types of cancer gene therapies, success stories, resources, and current clinical trials. ABOUT CANCER GENE THERAPY Enter here if you seek funding for cancer gene therapy research or wish to learn more about the research projects funded by ACGT. ABOUT RESEARCH GRANTS NEWS ITEMS ACGT mourns the loss of Dr. Judah Folkman, a founding member of our Scientific Advisory Council, who died at the young age of 74 in Denver of an apparent heart attack. The world has lost a wonderful human being and ACGT has lost a dear and special friend who inspired and guided us from inception. ACGT Meets Extensive Standards of America's Most Experienced Charity Evaluator: the BBB Wise Giving Alliance Subscribe for updates UPDATES ACGT Announces RFA for The Joan Miller and Linda Bernstein Gene Therapy Ovarian Cancer Award. Abstract Deadline: February 6, 2008 at 4pm E.S.T.

33. MMIA gene therapy. The link below contains an HTML version of the Microsoft PowerPoint used during the MMIA conference. gene therapy.htm. http://www.iupui.edu/~wellsctr/MMIA/htm/gene_therapy.htm

Gene Therapy The link below contains an HTML version of the Microsoft PowerPoint used during the MMIA conference. Gene Therapy.htm

34. Gene Therapy Currently, gene therapy is an experimental procedure that aims to correct only defective genes that cause disease and not other characteristics. http://www.biotechnologyonline.gov.au/human/genetherapy.cfm

Biotechnology Australia Biotechnology Online Current News Teaching Support ... Careers Gene therapy Genetic testing can reveal if a person has a genetic condition. Can we use biotechnology to cure them? allele - which may cause it to function less effectively or not at all. In time it may be possible to use gene therapy to replace an abnormal or faulty gene with a normal copy of the same gene. Currently, gene therapy is an experimental procedure that aims to correct only defective genes that cause disease and not other characteristics. In the future it may be used to treat ailments such as heart disease, inherited diseases or cancers. None of us are perfect Each human carries about half a dozen defective genes. Most of us do not suffer any harmful effects from our defective genes because we carry two copies of nearly all genes. Scientists are looking at gene therapy as a treatment for genetic disorders. This is a technique whereby the absent or faulty gene is replaced by a working gene, so that the body can make the correct enzyme or protein and consequently eliminate the root cause of the disease.

35. Alberta Gene Therapy Group A network of researchers looking to foster collaboration in several therapeutic fields. http://www.ualberta.ca/~britchie/agtg.html

Welcome to the Alberta Gene Therapy Group Home Page The Alberta Gene Therapy Group was born during a meeting organised by Lung-Ji Chang from the University of Alberta and Norman Wong from the University of Calgary held at the Holiday Inn, Red Deer, Alberta, November 23, Thursday, 1995. The Group is made up of people from the Unversity of Alberta , the Unversity of Calgary , the Alberta Cancer Board, the Alberta Heritage Foundation for Medical Research , the Alberta Research Council , the Alberta Science and Research Authority Alberta Health and Wellness , and MDS Health Ventures Ltd. They are all interested in research in the field of gene therapy. Contact information is provided below to foster collaboration. The group has been inactive recently. ... created by related sites Biochemistry , University of Alberta Department of Medical Microbiology and Immunology , University of Alberta Oncology , University of Alberta Department of Medical Genetics , University of Alberta Cell Biology , University of Alberta Membrane Proteins Research Group , University of Alberta Molecular and Cell Biology of Lipids , University of Alberta MRC Group in Protein Structure and Function , University of Alberta Protein Engineering Network of Centres of Excellence , University of Alberta Molecular Mechanisms of Growth Control , University of Alberta Institute for Biomolecular Design , University of Alberta Biochemistry and Molecular Biology , University of Calgary Cancer Biology Research Group , University of Calgary University of Alberta

36. Gene Therapy gene therapy is a type of biological therapy. There are a number of new types of treatments being researched that come under this general heading. http://www.cancerhelp.org.uk/help/default.asp?page=131

37. Harvard Gene Therapy Initiative gene therapy Initiative, Harvard Institute of Human Genetics, Harvard Medical School, Children s Hospital, Boston. Conducts research developing novel gene http://hgti.med.harvard.edu/

Gene Therapy Initiative Harvard Institute of Human Genetics HARVARD MEDICAL SCHOOL Dr. Richard Mulligan, Director The Harvard Gene Therapy Initiative was founded with the objective of promoting the use of gene therapy in both research and therapeutic applications and to conduct research developing new gene delivery vector technologies. Dr. Jeng-Shin Lee Associate Director Dr. Zhanyun Fan Manager 77 Avenue Louis Pasteur, HIM-412 Boston, MA 02115-5727 Phone: (617) 432-6465 , Please note: This is a temporary website. We will restore our full site as soon as possible. Thank you for your patience.

38. Technology Review: Gene Therapy For Alcoholics An interesting approach to curbing alcoholism is tested on rats. http://www.technologyreview.com/Biotech/20023/

Log in to My.TechnologyReview.com Register Home Infotech ... Events Wednesday, January 09, 2008 Gene Therapy for Alcoholics An interesting approach to curbing alcoholism is tested on rats. By Anna Davison Print E-mail Listen - Flash Listen - MP3 Subscribe to podcast What is this? Powered by Digg this Add to del.icio.us Add to Reddit Add to Facebook Slashdot It! Stumble It! Add to Newsvine Add to Connotea Add to CiteUlike Add to Furl Googlize this Add to Rojo Add to MyWeb Alcoholic animals: Researchers in Chile have used gene therapy to curb drinking in alcoholic rats. Credit: U.S. National Park Service Researchers in Chile have succeeded in keeping the drinking habits of alcoholic rats in check using gene therapy. The treatment mimics a natural mutation common in East Asian people, which lowers their tolerance to alcohol, making them less likely to become alcoholics. According to the National Institutes of Health, 17.6 million people abuse alcohol or are alcohol dependent in the United States alone. If the gene-therapy technique could be applied to humans, scientists say that it may be a valuable addition to the drugs and behavioral approaches currently used to treat alcoholism. The gene therapy works in a similar way to a drug currently used to treat alcoholics, which is effective but unpopular with patients, many of whom stop taking it.

39. GENE THERAPY This possibility, known as gene therapy, is only in its infancy. At present, no one knows how effective it will prove to be, even in the few conditions on http://www.srtp.org.uk/genthpy1.htm

SRT Home Page What is the SRT Project? What's New? Highlights ... The Big Issues The Big Issues "Engineering Genesis" Genetic Engineering GM Food GM Animals Human Genetics Cloning Xenotransplantation Patenting Risk Environment Climate Change Energy Nuclear Power Transport Eco-Congregation Church Energy Saving Technology Internet Issues SRT Publications SRT Newsletter SRT Information Sheets SRT Topical Papers ... Eco-Congregation S ociety, R eligion and T echnology P roject Church of Scotland Looking at the ethics of technology for a New Millennium Moral and Ethical Issues in Gene Therapy Dr Donald M.Bruce Contents Introduction Potential Ethical Issues Is it acceptable for human beings to manipulate human genes? Risk and the need for humility and prudence in genetic developments ... Links to other SRT Pages Introduction Potential Ethical Issues Perhaps the most basic underlying questions centre on a Christian understanding of the human being. What does this tell us vis a vis our genetic and physical makeup? What are therefore proper interventions into that genetic makeup? What would be improper in terms of our human dignity?

40. UK Cystic Fibrosis Gene Therapy Consortium A consortium whose aims are to provide cystic fibrosis gene therapy updates to researchers, medics, patients and their families. http://www.cfgenetherapy.org.uk/

Home Members Research Clinical Programme ... Vacancies About the Consortium The UK Cystic Fibrosis Gene Therapy Consortium (UK CFGTC) is the unified research programme of the three leading gene therapy groups in the UK. It was established in 2001 on the initiative of the Cystic Fibrosis Trust. The three member research groups are headed by Prof. David Porteous at the University of Edinburgh, Drs Deborah Gill and Steve Hyde at the University of Oxford and Prof. Eric Alton at Imperial College in London. Our Aim T he UK has always been at the forefront of gene therapy for cystic fibrosis (CF) and the members of the Consortium have already demonstrated proof-of-principle of correction of the underlying defect in the airways of CF subjects. The aim of the Consortium is to combine the intellectual and practical resources of the three groups and avoid duplication of efforts thereby enhancing progress towards clinically relevant gene therapy for CF. Communication The success of the Consortium, which consists of around 80 researchers spread over the three geographically distant sites, relies on regular communication between its members. All project leaders meet face-to-face every three months to present progress and prospectively discuss projects. These meetings are supplemented by smaller working groups (linking related projects) that communicate more often through phone-conferences, email and face to face to discuss more immediate practical issues. Core Facilities To ensure standardisation of results and high throughput, core facilities staffed by dedicated technicians have been set-up for the assessment of pre-clinical gene transfer.

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