21. European Society Of Gene Therapy esgt.org, European Society of gene therapy If your browser sticks here, click here gene therapy, esgt.org, health/medical, European Society of. http://www.esgt.org/

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22. CysticFibrosis.com Internet community for cystic fibrosis patients, families and loved ones. Information on clinical trials, gene therapy, testing, associations, research and events. http://cysticfibrosis.com/

Home CF Centers Associations Clinical Trials Gene Therapy ... New Design! Welcome to the newly designed CysticFibrosis.com website! comment on our forum... Through the eyes of CF I have learned through the years, that having insight and foresight are two valuable tools to have while surviving an illness. read more... Proper nutrition is vital I have learned through the years, that having insight and foresight are two valuable tools to have while surviving an illness. read more... Cystic Fibrosis Information CF is a genetic disorder that affects the respiratory, digestive and reproductive systems. Frequently Asked Questions While there are so many questions that are asked, some are left unanswered. We've got some answers to your FAQs! Just for kids! In terms that they can understand! Learn important information about daily living nutrition stories games ... snacks , and much more... CysticFibrosis.com latest Forum topics Families - Lots of questions, lots of fear Adults - Starting the xplant process, questions! ... Join our forums, it's compltely free! You may also post anonymously as well!

23. Gene Therapy - Wikipedia, The Free Encyclopedia gene therapy is the insertion of genes into an individual's cells and tissues to treat a hereditary diseases in particular. gene therapy typically aims to supplement a defective http://www.wikipedia.org/wiki/Gene_therapy

Main Page Recent changes Edit this page Page history ... Printable version Not logged in Log in Help Other languages: Dansk Deutsch Nederlands Gene therapy From 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 therapy typically aims to supplement a defective mutant allele with a functional one. Although the technology is still in its infancy, it has been used with some success. Antisense therapy is not strictly a form of gene therapy, but is often lumped together with them. Table of contents showTocToggle("show","hide") 1 Background 2 Types of gene therapy 2.1 Ex vivo 2.2 In vivo ... 6 External links Background In the 1980s, advances in molecular biology had already enabled human genes to be sequenced and cloned . Scientists looking for a method of easily producing proteins , such as the protein deficient in diabetics - insulin, investigated introducing human genes to bacterial DNA. The modified bacteria then produce the corresponding protein, which can be harvested and injected in people who cannot produce it naturally. Scientists took the logical step of trying to introduce genes straight into human cells, focusing on diseases caused by single-gene defects, such as

24. Frosst, Phyllis Ph.D Postdoctoral fellow with experience in gene therapy, cell biology and human genetics. http://www.scripps.edu/~phyllis/

25. Gene Therapy - An Overview gene therapy An Overview. BIO. " Biotechnology in Perspective." Washington, D.C. Biotechnology Industry Organization, 1990. Altered Genes. Each of us carries about half a dozen defective genes. The types of gene therapy described thus far all have one factor in common that is, that the tissues being treated are http://www.accessexcellence.org/AB/IWT/Gene_Therapy_Overview.html

Gene Therapy - An Overview BIO. "Biotechnology in Perspective." Washington, D.C.: Biotechnology Industry Organization, 1990. Altered Genes Each of us carries about half a dozen defective genes. We remain blissfully unaware of this fact unless we, or one of our close relatives, are amongst the many millions who suffer from a genetic disease. About one in ten people has, or will develop at some later stage, an inherited genetic disorder, and approximately 2,800 specific conditions are known to be caused by defects (mutations) in just one of the patient's genes. Some single gene disorders are quite common - cystic fibrosis is found in one out of every 2,500 babies born in the Western World - and in total, diseases that can be traced to single gene defects account for about 5% of all admissions to children's hospitals. In the U.S. and Europe, there are exciting new programs to 'map' the entire human genome - all of our genes. This work will enable scientists and doctors to understand the genes that control all diseases to which the human race is prone, and hopefully develop new therapies to treat and predict diseases. Diseases of Genetic Origin Most of us do not suffer any harmful effects from our defective genes because we carry two copies of nearly all genes, one derived from our mother and the other from our father. The only exceptions to this rule are the genes found on the male sex chromosomes. Males have one X and one Y chromosome, the former from the mother and the latter from the father, so each cell has only one copy of the genes on these chromosomes. In the majority of cases, one normal gene is sufficient to avoid all the symptoms of disease. If the potentially harmful gene is recessive, then its normal counterpart will carry out all the tasks assigned to both. Only if we inherit from our parents two copies of the same recessive gene will a disease develop.

26. Human Gene Therapy Human gene therapy. Some commentators on gene therapy have objected to any form of genetic manipulation, no matter how wellintentioned (VII, Rifkin 1983). http://www.georgetown.edu/research/nrcbl/scopenotes/sn24.html

SCOPE NOTE National Reference Center for Bioethics Literature The Joseph and Rose Kennedy Institute of Ethics Georgetown University Washington, DC 20057-1212 800-MED-ETHX; 202-687-3885; fax: 202-687-6770 e-mail: bioethics@georgetown.edu http://bioethics.georgetown.edu Human Gene Therapy Table of Contents I. Background II. Organizational Statements/Policies III. General IV. Philosophical Aspects ... Get a Quick Bib for Recent Literature on this Topic I. Background On September 14, 1990 researchers at the U.S. National Institutes of Health performed the first (approved) gene therapy procedure on four-year old Ashanti DeSilva. Born with a rare genetic disease called severe combined immune deficiency (SCID), she lacked a healthy immune system, and was vulnerable to every passing germ. Children with this illness usually develop overwhelming infections and rarely survive to adulthood; a common childhood illness like chickenpox is life-threatening. Ashanti led a cloistered existenceavoiding contact with people outside her family, remaining in the sterile environment of her home, and battling frequent illnesses with massive amounts of antibiotics. In Ashanti's gene therapy procedure, doctors removed white blood cells from the child's body, let the cells grow in the lab, inserted the missing gene into the cells, and then infused the genetically modified blood cells back into the patient's bloodstream. Laboratory tests have shown that the therapy strengthened Ashanti's immune system; she no longer has recurrent colds, she has been allowed to attend school, and she was immunized against whooping cough. This procedure was not a cure; the white blood cells treated genetically only work for a few months, and the process must be repeated every few months.

27. Leaders In Plasmid DNA Production, Recombinant Proteins And Vector Development NTC is a technologybased molecular biology and gene therapy service company, providing plasmid DNA manufacturing services, recombinant protein manufacturing http://www.natx.com/

Nature Technology Corporation Toll Free: 1 (888) 949-2264 Suitable for gene therapy and molecular biology and plasmid production.. Suitable for gene therapy and molecular biology and plasmid production.. Suitable for gene therapy and molecular biology and plasmid production.. Suitable for gene therapy and molecular biology and plasmid production.. SERVICES PDFs NTC's Technolgies Plasmid Technology Protein Technology DNA Vaccine Technology ... Databases and Sequences Analysis Tools WWW http://www.natx.com Licensing Plasmid DNA Proteins DNA Vaccines ... Corporate Profile

28. CNN.com - Gene Therapy Successful In Treating Hemophilia B, Researchers Say - Ma CNN http://www.cnn.com/2000/HEALTH/03/01/goodgene.therapy/index.html

health Editions myCNN Video ... Feedback CNN Sites CNN CNN Europe CNNfn CNNSI myCNN CNNfyi AllPolitics Languages Search CNN.com CNNSI.com CNNfn.com The Web HEALTH TOP STORIES New treatments hold out hope for breast cancer patients MORE TOP STORIES Thousands dead in India; quake toll rapidly rising Israelis, Palestinians make final push before Israeli election Davos protesters confront police MORE ... MORE MARKETS 4:30pm ET, 4/16 DJIA NAS SPORTS Jordan says farewell for the third time ... LOCAL EDITIONS: CNN.com Europe change default edition MULTIMEDIA: video video archive audio multimedia showcase ... more services E-MAIL: Subscribe to one of our news e-mail lists Enter your address: DISCUSSION: chat feedback CNN WEB SITES: CNNfyi.com CNN.com Europe AsiaNow Spanish ... Korean Headlines TIME INC. SITES: Go To ... Time.com People Money Fortune EW CNN NETWORKS: CNN anchors transcripts Turner distribution SITE INFO: help contents search ad info ... jobs WEB SERVICES: Gene therapy successful in treating hemophilia B, researchers say March 1, 2000 Web posted at: 10:45 p.m. EST (0345 GMT) (CNN) Researchers at the Children's Hospital of Philadelphia and Stanford University have successfully used gene therapy to treat hemophilia B, according to a study published in this month's Nature Genetics.

29. CNN - FDA To Defend Closure Of Gene Therapy Trials To Senate Subcommittee - Febr CNN http://www.cnn.com/2000/HEALTH/02/02/gene.therapy.01/index.html

[an error occurred while processing this directive] MAIN PAGE WORLD U.S. LOCAL ... daily almanac MULTIMEDIA: video video archive audio multimedia showcase ... more services E-MAIL: Subscribe to one of our news e-mail lists. Enter your address: document.write(' '); Or: Get a free e-mail account E-MAIL DISCUSSION: message boards chat feedback CNN WEB SITES: AsiaNow Svenska Norge Danmark ... Italian FASTER ACCESS: europe japan TIME INC. SITES: Go To ... Time.com People Money Fortune EW CNN NETWORKS: more networks transcripts SITE INFO: help contents search ad info ... jobs WEB SERVICES: health > story page FDA to defend closure of gene therapy trials to Senate subcommittee February 2, 2000 Web posted at: 10:20 a.m. EST (1520 GMT) From Medical Correspondent Elizabeth Cohen (CNN) A Senate subcommittee will be hearing testimony Wednesday into whether the Food and Drug Administration did the right thing in shutting down gene therapy trials at the University of Pennsylvania after a patient's death. Eighteen-year-old Jesse Gelsinger of Tucson, Arizona, died in September after receiving an experimental gene therapy at the University of Pennsylvania for an inherited liver disease. On January 21, the FDA, citing numerous research regulation violations, shut down gene therapy and other clinical trials at the University of Pennsylvania.

30. CNN.com - Gene Therapy For Cancer? - August 1, 2000 CNN http://www.cnn.com/2000/HEALTH/cancer/08/01/cancer.gene.therapy/index.html

cancer Editions myCNN Video ... Feedback CNN Sites CNN CNN Europe CNNfn CNNSI myCNN CNNfyi AllPolitics Languages Search CNN.com CNNSI.com CNNfn.com The Web HEALTH TOP STORIES New treatments hold out hope for breast cancer patients MORE TOP STORIES Thousands dead in India; quake toll rapidly rising Israelis, Palestinians make final push before Israeli election Davos protesters confront police MORE ... MORE MARKETS 4:30pm ET, 4/16 DJIA NAS SPORTS Jordan says farewell for the third time ... LOCAL EDITIONS: CNN.com Europe change default edition MULTIMEDIA: video video archive audio multimedia showcase ... more services E-MAIL: Subscribe to one of our news e-mail lists Enter your address: DISCUSSION: chat feedback CNN WEB SITES: CNNfyi.com CNN.com Europe AsiaNow Spanish ... Korean Headlines TIME INC. SITES: Go To ... Time.com People Money Fortune EW CNN NETWORKS: CNN anchors transcripts Turner distribution SITE INFO: help contents search ad info ... jobs WEB SERVICES: Overview Genome guide Glossary Related sites ... Video Archive Gene therapy for cancer? Early results promising August 1, 2000 Web posted at: 12:30 p.m. EDT (1630 GMT) From staff and wire reports (CNN) Researchers have found evidence that gene therapy may be an effective cancer treatment.

31. Gene Therapy - An Overview gene therapy An Overview. BIO. Biotechnology in Perspective. Washington, DC Biotechnology Industry Organization, 1990. Altered Genes. gene therapy. http://www.accessexcellence.org/AB/BA/Gene_Therapy_Overview.html

Gene Therapy - An Overview BIO. "Biotechnology in Perspective." Washington, D.C.: Biotechnology Industry Organization, 1990. Altered Genes Each of us carries about half a dozen defective genes. We remain blissfully unaware of this fact unless we, or one of our close relatives, are amongst the many millions who suffer from a genetic disease. About one in ten people has, or will develop at some later stage, an inherited genetic disorder, and approximately 2,800 specific conditions are known to be caused by defects (mutations) in just one of the patient's genes. Some single gene disorders are quite common - cystic fibrosis is found in one out of every 2,500 babies born in the Western World - and in total, diseases that can be traced to single gene defects account for about 5% of all admissions to children's hospitals. In the U.S. and Europe, there are exciting new programs to 'map' the entire human genome - all of our genes. This work will enable scientists and doctors to understand the genes that control all diseases to which the human race is prone, and hopefully develop new therapies to treat and predict diseases. Diseases of Genetic Origin Most of us do not suffer any harmful effects from our defective genes because we carry two copies of nearly all genes, one derived from our mother and the other from our father. The only exceptions to this rule are the genes found on the male sex chromosomes. Males have one X and one Y chromosome, the former from the mother and the latter from the father, so each cell has only one copy of the genes on these chromosomes. In the majority of cases, one normal gene is sufficient to avoid all the symptoms of disease. If the potentially harmful gene is recessive, then its normal counterpart will carry out all the tasks assigned to both. Only if we inherit from our parents two copies of the same recessive gene will a disease develop.

32. Laboratory Of Gene Therapy Research - Rigshospitalet Focusing on C/EBPa in growth arrest, differentiation and cancer, and nonsensemediated mRNA decay in development, RNA traffic and disease, at Copenhagen University Hospital, Denmark. http://www.labgenterapi.dk

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33. Cancer Gene Therapy Supported by the International Society for Cancer gene therapy, this resource publishes original research and hosts diverse views. http://www.naturesj.com/cgt/index.html

nature.com about npg nature science update naturejobs ... site index SEARCH my account e-alerts subscribe register ... Journal home For readers Content Advance online publication Online sample issue E-alerts ... Society publishing NPG Subject areas Access material from all our publications in your subject area: Biotechnology Cancer Chemistry Clinical Medicine ... Microbiology NEW! Molecular Cell Biology Neuroscience Pharmacology Physics ISSN 0929-1903 2004 Volume 11 Publishes 12 issues a year ISI 2002 Impact Factor 2.929 Rank 42/115 Genetics and Heredity, 32/114 Oncology, View tables of contents The essential gene therapy resource for cancer researchers and clinicians Pertinent and practical, the Journal keeps its readers up to date with the latest developments in gene therapy for cancer Presents diverse viewpoints from academic, industry, and regulatory perspectives Publishes original laboratory and clinical research papers, case reports and review articles Topics covered include: antisense technology; drug resistance; hematopoietic gene transfer; homologous recombination; ribozyme technology; tumor immunotherapy; tumor suppressors

34. Ingenta Select www.catchword.com/rpsv/catchword/mal/10430342/contp11.htm gene therapy for X-linked severe combined immune deficiency(X-SCID http://www.catchword.com/titles/10430342.htm

Human Gene Therapy Human Gene Therapy is available online via Ingenta Select. There are currently 96 issues available for viewing. You can browse the contents pages of online issues free of charge. Please note that, in order to view the full text of online articles, you will need to follow the steps below. Accessing Full Text Documents If you, or your institution, have an online subscription to Human Gene Therapy then please browse the volumes and issues available To enable online subscriptions you need to quote your CatchWord Identification number ("CID") to your Subscription Agent or to the Publisher. If you do not have a CID, please register online The Current Issue is always free The May 1st 2004 issue is available free of charge. Table of Contents Alerting Service If you would like to receive an email notification of the Table of Contents for each new online issue of Human Gene Therapy please enter your email address below: E-mail address:

35. The International Symposium On Molecular Diagnostics Skin Gene Therapy Topics include chip technology and proteomics, targeting strategies, skin gene transfer, updates on vector systems, and inhibiting viral systems. March 27 29, 2003, D¼sseldorf, Germany. http://www.isgt-congress.org/

Home ISGT 2003 Keynote Speaker Program Downloads ... Contact Congress Management Prof. Dr. med. Ulrich Hengge Heinrich-Heine-University Dept. of Dermatology Moorenstrasse 5 40212 Düsseldorf GERMANY Phone: +49(0)211/ 811 8066 Fax: +49(0)211 / 811 8700 ulrich.hengge@isgt-congress.org You can contact the congress team per e-mail: General information: info@isgt-congress.org Registration, cancellation, replacements: registration@isgt-congress.org Newsletter, conference update: newsletter@isgt-congress.org press@isgt-congress.org Hotel reservation Lindner Hotel info.congresshotel@lindner.de Technical problems, web site issues: webmaster@isgt-congress.org GET The 2003 PROGRAM WHO SHOULD ATTEND Researcher and Scientists Medical Professionals Bioinformatics Pharmaceutical Officers Regulators Journalists Students (c) 2003, Prof. Dr. Ulrich Hengge, Uni Duesseldorf

36. UNC Gene Therapy Center The UNC gene therapy Center utilizes the adenoassociated virus (AAV) in the quest to develop an efficient viral vector system for use in human gene therapy. http://www.med.unc.edu/wrkunits/3ctrpgm/genether/welcome.htm

Click here to learn more about our gene therapy research. Announcements 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 Stilwell J and Samulski RJ. 2004. Role of Viral Vectors and Virion Shells on Cellular Gene Expression . Molecular Therapy, 9(3): 337-346. Fall 2003 - The UNC Gene Therapy Center was featured in Endeavors Magazine for our work with Canavan Disease. Click here to learn more and read the article New Publications Rabinowitz JE, Bowles DE, Faust SM, Ledford JG, Cunningham SE, Samulski RJ. 2004.

37. Carl C. Icahn Center For Gene Therapy And Molecular Medicine Anticipating new horizons of biomedical research, our department was established in 1996 at Mount Sinai School of Medicine to promote and accelerate the science and technology of gene/cellular therapeutics. http://www.genetherapy-mssm.net/home.php

Wednesday May 26, 2004 WORK IN PROGRESS SERIES

38. CBER - Cellular & Gene Therapy Cellular gene therapy. The Center for Biologics FDA has not yet approved any human gene therapy product for sale. However, the amount of http://www.fda.gov/cber/gene.htm

FDA Home Page CBER A-Z Index CBER Search Contact CBER ... CBER Home Page The Center for Biologics Evaluation and Research (CBER) regulates human gene therapy products - products that introduce genetic material into the body to replace faulty or missing genetic material, thus treating or curing a disease or abnormal medical condition. CBER uses both the Public Health Service Act and the Federal Food Drug and Cosmetic Act as enabling statutes for oversight. FDA has not yet approved any human gene therapy product for sale. However, the amount of gene-related research and development occurring in the United States continues to grow at a fast rate and FDA is actively involved in overseeing this activity. FDA has received many requests from medical researchers and manufacturers to study gene therapy and to develop gene therapy products. Such research could lead to gene-based treatments for cancer, cystic fibrosis, heart disease, hemophilia, wounds, infectious diseases such as AIDS, and graft-versus-host disease. Features Cellular Therapy: Potential Treatment for Heart Disease New Human Gene Transfer Research Data System Type I Diabetes / Pancreatic Islet Transplantation Use of Cloning Technology to Clone a Human Being Topics Biol Resp Modifiers Adv Comm Publications Warning Letters Xenotransplantation Action Plan ... GT Patient Tracking System Updated March 26, 2004

39. Licensing Medical Implanting Technology To Biotech And Pharma Companies Implants for pharmaceutical and gene therapy controlled release pellets, brachytherapy seeds, and chip and nanodevices. http://www.NjeX.com/

NjeX Implant Introducing Systems for the Future's Medical Implant Devices Convenient and Cost-Effective Implanting of: Controlled Release Drug/Gene Therapy Devices Various Chip and Nano-Device Implants Radionuclide and Thermo-Active Seeds, or... Your New Implant Device? Privacy, terms and conditions

40. Fundamentals Of Gene Therapy Fundamentals of gene therapy. You look a little like your mother and a little like your father because of the genes they gave to you. 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

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