61. Gene Therapy In an attempt to cure the children—who would otherwise die at a young age—physicians used gene therapy to provide them with normal gammac genes. http://www.sumanasinc.com/scienceinfocus/sif_genetherapy.html

Click to view animation Gene Therapy This particular trial has had striking success as well as tragedy. Eight of the eleven children are currently thriving. However, in two cases the therapy successfully introduced gamma-c genes, but these children have since developed leukemia. In both children, a gamma-c gene inserted next to another gene, called . The gene has previously been linked to leukemia, and scientists speculate that the insertion of the gamma-c gene next to may have overstimulated the gene, causing T cells to proliferate in excess. An effect, in combination with the proliferation-inducing effects of the gamma-c gene itself, may be the cause of the leukemia in these two patients. Scientists are still investigating other possible causes. From this single trial, it is clear that gene therapy holds significant promise, yet it is also clear that it poses significant risks. To learn more about the application of gene therapy in SCID, view the accompanying animation. Links The first set of results for this gene therapy trial was published in the journal Science Go to article...

62. Neurologix Gene Therapy Approach To Parkinson's Disease Granted Fast Track Desig Neurologix, Inc. (OTCBBNRGX), a biotech company engaged in the research and development of innovative gene therapies for the brain and central nervous http://www.medicalnewstoday.com/articles/91995.php

@import "/css/pagelayout.css"; @import "/css/default.css"; @import "/css/defaultnews.css"; health news health videos opinions forum contact ... News Archive Web Wikipedia Medical Dictionary [ link Parkinson's Disease News Useful Links Video Library Neurologix Gene Therapy Approach To Parkinson's Disease Granted Fast Track Designation From FDA Main Category: Parkinson's Disease Also Included In: Neurology / Neuroscience Article Date: 17 Dec 2007 - 3:00 PST email to a friend printer friendly view / write opinions rate article newsletters Visitor Ratings: Healthcare Professional: General Public: >> rate this article Neurologix, Inc. (OTCBB:NRGX), a biotech company engaged in the research and development of innovative gene therapies for the brain and central nervous system, announced that the U.S. Food and Drug Administration has granted Fast Track Designation for the company's experimental gene transfer procedure for the treatment of advanced Parkinson's disease. The Neurologix procedure delivers a gene (glutamic acid decarboxylase, or GAD) to the subthalamic nucleus of the brain, where it makes an inhibitory neurotransmitter called GABA that helps to quiet the abnormal brain activity that is correlated with motor deficits characterizing Parkinson's disease. Results of a Phase 1 clinical study showed that the Neurologix gene transfer procedure was both well tolerated and resulted in improved motor function and brain metabolism for patients with advanced Parkinson's disease over the course of the one-year study. Neurologix is currently preparing to initiate a Phase 2 study of its Parkinson's disease treatment by early 2008, subject to final FDA consent to the study protocol.

63. Gene Therapy The treatment of a disease by introducing DNA into the cells of the patient. http://genome.wellcome.ac.uk/doc_wtd020911.html

var g_HttpRelativeWebRoot = "/stellent/"; var SSContributor = false; var SSForceContributor = false; var SSHideContributorUI = false; var ssUrlPrefix = "/Genome/"; g_HttpRelativeWebRoot = "/stellent/";SSContributor = false; About this site Sitemap Contact us In the genome ... Background Gene therapy Developing therapies News Features Background ... Treating disease Gene therapy 27/8/02. By Richard Twyman The treatment of a disease by introducing DNA into the cells of the patient. There are several types of gene therapy: Gene augmentation therapy This is appropriate for the treatment of inherited disorders caused by the loss of a functional gene product. The aim is to add a functional copy of the lost gene back into the genome and express it at sufficient levels to replace the missing protein. It is only suitable if the pathogenic effects of the disease are reversible. Gene inhibition therapy This is suitable for the treatment of infectious diseases, cancer and inherited disorders caused by inappropriate gene activity. The aim is to introduce a gene whose product inhibits the expression of the pathogenic gene or interferes with the activity of its product. Killing of specific cells This is suitable for diseases such as cancer that can be cured by eliminating certain populations of cells. The aim is to express within such cells a suicide gene, whose product is toxic. One approach is the expression of an enzyme that converts a harmless prodrug into a highly toxic molecule. Another is the expression of a protein that makes the cells vulnerable to attack by the immune system. It is very important to ensure that suicide genes are appropriately targeted, otherwise the therapy would result in widespread cell death.

64. BioPortal | Gene Therapy What is gene therapy?Genetic DiseasesTypes of gene therapyThe Science – How does gene therapy Work?Gene of the Moment p53Biotechnology and Gene http://biobasics.gc.ca/english/View.asp?x=779

65. Program In Human Gene Therapy Facilitates the application of basic and preclinical research to the treatment of human disease through vector development and gene discovery and http://medicine.ucsd.edu/gt/

THE CELLULAR AND MOLECULAR MEDICINE - EAST building Located in the UCSD School of Medicine and home to the Program in Human Gene Therapy Welcome to the UCSD School of Medicine's Program in Human Gene Therapy. Our goals are to: Catalyze basic research into the genetics and cell biology of gene transfer Facilitate the application of basic and pre-clinical research to the treatment of human disease The Program includes a Vector Development core laboratory . Functions include: The preparation of research-grade gene transfer materials, including the production of retroviral, lentiviral, adenoviral, adeno-associated and herpes viral vectors Assistance at all stages of vector development, including advice on the design of vector constructs, plasmid preparation, production of virus, and transduction of cells. For additional information, link to the following Program Pages: Vector Development Laboratory Product Safety Information Seminar Series Information Contact the Program Other UCSD Sites: UCSD Homepage UCSD School of Medicine This page was updated April 18, 2007. For questions about this page contact: Dr. Miyanohara

66. Gene Therapy In Alcoholic Rats Scientists at Brookhaven National Laboratory have shown that increasing the level of a brain protein important for transmitting pleasure signals can turn http://www.bnl.gov/bnlweb/pubaf/pr/2001/bnlpr090501.htm

For more information, contact: Karen McNulty Walsh, 631 344-8350, or Mona S. Rowe, 631 344-5056 Sept. 9, 2001 Gene Therapy Reduces Drinking in "Alcoholic" Rats UPTON, NY - Scientists at the U.S. Department of Energy's Brookhaven National Laboratory have shown that increasing the level of a brain protein important for transmitting pleasure signals can turn rats that prefer alcohol into light drinkers, and those with no preference into near teetotalers. The findings, published in the first September 2001 issue of the Journal of Neurochemistry (Vol. 78, No. 5), may have implications for the prevention and treatment of alcoholism in humans. "This is a preliminary study, but when you see a rat that chooses to drink 80 to 90 percent of its daily fluid as alcohol, and then three days later it's down to 20 percent, that's a dramatic drop in alcohol intake - a very clear change in behavior," said Panayotis Thanos , the lead researcher. "This gives us great hope that we can refine this treatment for future clinical use."

67. UMHS Center For Gene Therapy The center links basic science, clinical investigation and technology transfer endeavors in the area of gene therapy, while also serving as a resource for http://www.med.umich.edu/cgt/

University of Michigan Medical System Center for Gene Therapy Search the CGT Website Resources Seminar Series Membership Application Intramural Grant Program ... Search Site Updated 10/21/2004 Mission The Center for Gene Therapy at the University of Michigan Medical Center was created to link basic science, clinical investigation and technology transfer. The Center fosters a multidisciplinary approach to new research as well as collaborative research endeavors in the area of gene therapy; extends the services of existing and newly created research cores to investigators; and serves as a resource for information and education. Scope of Activities To aid medical researchers, we have divided the Center into four programs. Each program offers products and services to aid the medical researcher here at Michigan and elsewhere. The products and services are : Gene Vector Program The Vector Core Laboratory Clinical Trials Program Regulatory Application Assistance Educational Program Administrative Program Cellular Therapy Development Grant Program Background The University of Michigan is an established leader in the field of gene therapy and molecular medicine and has pioneered a number of the most innovative gene therapy techniques to be tested in the United States.

68. Genlantis Reagents For Transfection; DNA, RNAi, SiRNA Terms Conditions. . Privacy Policy. . Careers. . @ 2007 Genlantis / a division of gene therapy Systems, Inc. RNA Interference. . Cell Culture Reagents http://www.genlantis.com/

Search Citation Search Shopping Cart Genlantis is focused on developing innovative reagents for transfection, gene expression, RNA interference, primary cell culture, and sample preparation in proteomics research. We are dedicated to serving the needs of science researchers worldwide by embracing innovation. Quick Links Transfection center Explorer newsletter Frequently asked questions Literature request Receive a $50 Starbucks Gift Card when purchasing a 1,000 reaction kit. Featured Products GeneSilencer Reagent Efficient transfection of siRNA into mammalian cells. more info TurboCells Competent E. coli E. coli transformation in 3 Minutes. more info TrojanPORTER Reagent High Speed, High Efficiency, Low Toxicity Transfection of DNA more info Site Map Careers Genlantis / a division of Gene Therapy Systems, Inc. RNA Interference Cell Culture Reagents Competent Cells Human Cells ... siRNA Transfection

69. Pittsburgh Human Gene Therapy Center The mission of the The Pittsburgh Human gene therapy Center is to foster the growth and development of multidisciplinary preclinical and clinical research http://www.pitt.edu/~rsup/phgt/index.html

This site is now incorporated within The Molecular Medicine Institute. Please Click Here

70. Blood -- Collected Resources : Gene Therapy Selecting highly affine and wellexpressed TCRs for gene therapy of Hematopoietic stem-cell gene therapy of hemophilia A incorporating a porcine factor http://bloodjournal.hematologylibrary.org/cgi/collection/gene_therapy

window.onload=preloadImages; SEARCH: Advanced Gene Therapy Citations 1-10 of 382 total displayed. Most recent content (15 Dec 2007): Leszek Lisowski and Michel Sadelain Blood 2007; 110: 4175-4178. [Abstract] [Full text] [PDF] Past content (since Aug 1998): Deletions within the HSV-tk transgene in long-lasting circulating gene-modified T cells infused with a hematopoietic graft Blood 2007; 110: 3842-3852. [Abstract] [Full text] [PDF] Selecting highly affine and well-expressed TCRs for gene therapy of melanoma Annelies Jorritsma, Raquel Gomez-Eerland, Maarten Dokter, Willeke van de Kasteele, Yvonne M. Zoet, Ilias I. N. Doxiadis, Nathalie Rufer, Pedro Romero, Richard A. Morgan, Ton N. M. Schumacher, and John B. A. G. Haanen Blood 2007; 110: 3564-3572. [Abstract] [Full text] [PDF] Co-expression of cytokine and suicide genes to enhance the activity and safety of tumor-specific cytotoxic T lymphocytes Concetta Quintarelli, Juan F. Vera, Barbara Savoldo, Greta M. P. Giordano Attianese, Martin Pule, Aaron E. Foster, Helen E. Heslop, Cliona M. Rooney, Malcolm K. Brenner, and Gianpietro Dotti

71. The Children's Hospital At Westmead - Professionals - Gene Therapy Research Unit A joint initiative of The New Children s Hospital and the Children s Medical Research Institute is undertaking gene therapy clinical trials in Australia. http://www.chw.edu.au/prof/services/genetherapy/

feedback sitemap gap for health telehealth ... Gene Therapy Research Unit Gene Therapy Research Unit A joint initiative of The Children's Hospital at Westmead and Children's Medical Research Institute Main Projects Publications Staff Contact Introduction The ultimate aim of biomedical research is to improve human health, but its more immediate impact, beyond the accumulation of biological knowledge, is an increase in our capacity to diagnose disease. Therapeutic benefits follow, but significantly more slowly. Herein lies a potentially frustrating paradox. When biomedical knowledge is undergoing rapid growth (as it is at present) we can anticipate a widening of the gap between our capacity to diagnose and our capacity to treat disease. For this reason it is fundamentally important that increased effort be focused on realising the therapeutic potential of advances in biological knowledge. Such an effort demands that we explore entirely new therapeutic paradigms and surmount the formidable translational challenges inherent in taking new therapies from the laboratory bench to the patient bedside. Gene Therapy, or "the use of genes as medicine" is one such paradigm, with immense but largely unrealised potential. The Gene Therapy Research Unit, a joint initiative of the The Children's Hospital at Westmead and

72. International Society For Cell & Gene Therapy Of Cancer Home Page International Society for Cell gene therapy of Cancer. http://www.iscgt.org.uk/

ISCGT) HOME NEWS LINKS EVENTS ... Membership ISCGT Home Welcome to the ISCGT Web! Our mission is to become a truly International Society for clinicians and scientists alike. New visitors are recommended to visit About ISCGT first. Register here for Society Membership Are you aware of member privileges? click here ISCGT links Tell us about your site! ISCGT Mumbai, India 16-18 November 2007 Click here to be redirected to the ISCGT India website Sidney Kimmel Cancer Centre (SKCC) presents the CME Conference 'New Targets and Delivery Systems in Cancer Diagnosis and Treatment' ... 13-15 October 2006 Reviews by speakers at the ISCGT meeting may be found at: http://www.bioscience.org/current/special/tagawa.htm ISCGT Shenzhen, China 9-11 December 2005 ... To view the meeting report please click here ISCGT Secretariat : iscgt@kcl.ac.uk

73. Center For Gene Therapy Of Cystic Fibrosis And Other Genetic Diseases The Iowa Center for gene therapy was established in 1998 to promote research and training in molecular medicine with a focus on translational research using http://genetherapy.genetics.uiowa.edu/

74. Circulation Research -- Subspecialty Collections : Gene Therapy gene therapy to Inhibit the Calcium Channel ß Subunit Physiological Consequences and Pathophysiological Effects in Models of Cardiac Hypertrophy Eugenio http://circres.ahajournals.org/cgi/collection/gene_ther

Search: Advanced Search Circulation Research Home Subscriptions Archives ... AHA Journals Home Gene therapy Citations 1-10 of 141 total displayed. Most recent content (4 Jan 2008): Integrative Physiology Prevention of Dystrophin-Deficient Cardiomyopathy in Twenty-One-Month-Old Carrier Mice by Mosaic Dystrophin Expression or Complementary Dystrophin/Utrophin Expression Brian Bostick, Yongping Yue, Chun Long, and Dongsheng Duan Circ. Res. 102: 121 -130; published online before print as doi:10.1161/CIRCRESAHA.107.162982 [Abstract] [Full text] Past content (since Jan 1999): Reviews Emerging Role of MicroRNAs in Cardiovascular Biology Michael V.G. Latronico, Daniele Catalucci, and Gianluigi Condorelli Circ. Res. 2007; 101: 1225-1236, doi:10.1161/CIRCRESAHA.107.163147 [Abstract] [Full text] Integrative Physiology Regression of Abdominal Aortic Aneurysms by Simultaneous Inhibition of Nuclear Factor B and Ets in a Rabbit Model Takashi Miyake, Motokuni Aoki, Hisao Masaki, Tomio Kawasaki, Masako Oishi, Kazusaburo Kataoka, Toshio Ogihara, Yasufumi Kaneda, and Ryuichi Morishita Circ. Res. 101: 1175 -1184; published online before print as doi:10.1161/CIRCRESAHA.107.148668

75. Vecura.com - Gene Therapy Vectors Vecura is a contract manufacturer of gene therapy vectors and cell therapy products for clinical trials. The laboratory is a part of the gene therapy Center http://www.vecura.com/

Vecura is a contract manufacturer of gene therapy vectors and cell therapy products for clinical trials. The laboratory is a part of the Gene Therapy Center at Karolinska University Hospital. Karolinska University Hospital is affiliated with Karolinska Institutet Click here to enter

76. Genes And HIV - The Body Scientists Make Progress Toward gene therapy for HIV Infection (July 1, 1998) gene therapy That Prolongs CD4+ T Cell Survival In HIVInfected http://www.thebody.com/index/treat/genether.html

@import url(/css/thebody.css); @import url(/css/subtopic.css); @import url(/css/treatment.css); var zflag_nid="362"; var zflag_cid="1"; var zflag_sid="0"; var zflag_width="728"; var zflag_height="102"; var zflag_sz="26"; Entire Site Articles/Fact Sheets Ask the Experts Conference Coverage New HIV Medication Approved: HIV/AIDS Basics HIV Prevention HIV Testing ... More ... Genes and HIV Study Finds Genetic Influence on Pace of HIV/AIDS Progression (October 22, 2007) From U.S. National Institute of Allergy and Infectious Diseases Biotech Firm Creates Genetically Modified Immune Cells That Resist HIV Infection in Vitro (December 20, 2005) Sangamo aims to modify patients' immune cells to possess a CCR5 receptor mutation that may make them "uninfectable" In Kaiser Daily HIV/AIDS Report , from Henry J. Kaiser Family Foundation California: Scientists Tout Stem Cells' Use to Battle HIV (November 3, 2005) In CDC HIV/Hepatitis/STD/TB Prevention News Update , from U.S. Centers for Disease Control and Prevention The Development Pipeline for Cytokines, Immunomodulators and Gene Therapy (September 29, 2005) In TAGline , from Treatment Action Group Human Cells Can "Silence" HIV Genes (May 19, 2005)

77. Gene Therapy Sites A biopharmaceutical company based in Carlsbad, California, with technologies in four core areas HIV, autoimmune disease, gene therapy and cancer . http://www.uq.edu.au/vdu/GeneTherapylinks.htm

Virology The Basics Taxonomy Links ... HOME Headlines DNA Down Under New section on RNA interference as a tool to block virus replication. Gene Therapy Sites General Information Labs/Centres/Universities/Societies Projects/Personal Sites Site Libraries News Clippings Regulatory Bodies Conventions and Conferences Journals and Books Commercial Interests General Information AAV Vectors Clinical Trials Database From the Journal of Gene Medicine. Gene Information Technology Unit To foster a more favourable environment for the products of gene technology by providing clear, accurate and unbiased information. Gene Therapy - An Overview From Biotech Applied Gene Therapy: Potential, Pitfalls and Prospects By Inder M. Verma for an NIH Director's Wednesday Afternoon Lecture (WAL) Human Genome Project Information - Gene Therapy Introduction to Gene Therapy Provides access to information presented in a course here at Vanderbilt Univerity Medical Center Lentiviruses/HIV: Pathogenesis Relevant to new lentivirus vectors

78. Gene Therapy At the forefront of medicine, gene therapy brings you the latest research into genetic and cellbased technologies and the development of clinical methods http://www.ovid.com/site/catalog/Journal/1270.jsp?top=2&mid=3&bottom=7&subsectio

79. Oxford University Gene Medicine. UK CFGTC Cystic Fibrosis gene therapy Oxford University GeneMedicine. Nonviral gene therapy for the lung disease cystic fibrosis. Nuffield Department of Clinical http://users.ox.ac.uk/~genemed/

80. Virus Vectors & Gene Therapy Virus Vectors and gene therapy A review by David Peel, University of Leicester. http://www.tulane.edu/~dmsander/WWW/335/peel/peel1.html

David Peel MBChB Special Study Module Project Report University of Leicester. Introduction: Viruses as Vectors Retrovirus Vectors Adenovirus Vectors Adeno-Associated Virus ... References Introduction: Snyder et al, 1997 ), whereby the functional form of the gene would be added to the cell restoring it to a normal phenotype. However, research is underway to treat monogenic dominantly inherited diseases such as hypercholesteroleamia ( ). Regulation of cellular proliferation e.g. to prevent atherosclerosis following angioplasty ( Kim et al, 1997 ), or to promote of cellular repair following trauma to the CNS ( Federoff et al, 1992 ) are also currently being investigated. Viruses as Vectors: ^TOP^ Next Section University of Leicester

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