61. Harvard Gene Therapy Initiative Provides plasmid construction and virus production services for gene therapy in both research and Category Science Biology Animal Mammal Human gene therapy......gene therapy Initiative, Harvard Institute of Human Genetics, HarvardMedical School, Children's Hospital, Boston. Conducts research 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. John Gray Plasmid Construction Dr. Jeng-Shin Lee Virus Production 77 Avenue Louis Pasteur, HIM-412 Boston, MA 02115-5727 Phone: (617) 432-1249 , Fax: (617) 432-7044 E-mail contact: gray@hihg.med.harvard.edu Website liaison: alorch@hms.harvard.edu

62. Gene Therapy For The Bald Forget the Hair Club for Men. gene therapy may someday help bald men to grow new hair. Scientists graft tiny bits of human scalp to mice in an effort to develop a method of delivery of genetic material to hair follicles. Wired News http://www.wired.com/news/news/story/20142.html

63. FDA Places Temporary Halt On Gene Therapy Trials Using Retroviral Vectors In Blo FDA Places Temporary Halt on gene therapy Trials Using RetroviralVectors in Blood Stem Cells. FDA Talk Papers are prepared by the http://www.fda.gov/bbs/topics/ANSWERS/2003/ANS01190.html

FDA Talk Papers are prepared by the Press Office to guide FDA personnel in responding with consistency and accuracy to questions from the public on subjects of current interest. Talk Papers are subject to change as more information becomes available. Media Inquiries: 301-827-6242 January 14, 2003 Consumer Inquiries: 888-INFO-FDA FDA PLACES TEMPORARY HALT ON GENE THERAPY TRIALS USING RETROVIRAL VECTORS IN BLOOD STEM CELLS Agency Action Provides Way for Patients to Continue Therapy Under Certain Circumstances if Additional Measures Taken In a precautionary measure, the Food and Drug Administration (FDA) today placed on "clinical hold" all active gene therapy trials using retroviral vectors to insert genes into blood stem cells. FDA took this action after it learned that a second child treated in a French gene therapy trial has developed a leukemia-like condition. Both this child, and another who had developed a similar condition last August, had been successfully treated by gene therapy for X-linked severe combined immunodeficiency disease (X-SCID), also known as "bubble baby syndrome." Infants with X-SCID have a gene defect that leads to a complete lack of white blood cells that can fight infection. Without treatment, they die from complications of infectious diseases during the first year of life. The only treatment for this condition is a bone marrow transplant.

64. Coming Soon! Develops new methods and applications of human gene therapy for cancer prevention, treatment and cure; Category Science Biology Animal Mammal Human gene therapy......The site you requested is currently unavaliable. It will be functioning shortly,so check back often! Go back, or return to Spindustry Systems, Inc. http://www.hgtri.org/

The site you requested is currently unavaliable. It will be functioning shortly, so check back often! Go back , or return to Spindustry Systems, Inc. Please report all problems to webmaster

65. CBER - Cellular & Gene Therapy The Center for Biologics Evaluation and Research (CBER) regulates human gene therapyproducts products that introduce genetic material into the body to http://www.fda.gov/cber/gene.htm

Blood Therapeutics Vaccines Allergenics ... About Us 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. Last Updated: 1/24/2003 Features The Sixth US-Japan Cellular and Gene Therapy Conference - 2/20/2003 Cloning Technology As a consequence of scientific and biotechnological progress during the past decades, new biological therapies involving somatic cells and genetic material are being investigated.

66. IHGT - Institute For Human Gene Therapy Institute for Human gene therapy, University of Pennsylvania. Providing a foundation for basic research necessary to assure the success of human gene therapy. Main page. http://www.med.upenn.edu/~ihgt/

As of September 13th, 2002, the Institute of Human Gene Therapy (IHGT) web site has been permenantly removed. No forwarding or contact information exists at this time. Please update your bookmarks

67. FINNISH GENE THERAPY SOCIETY - MAIN PAGE gene therapy has matured as an exciting new field of research which will havea significant impact on biology and medicine during the next decade. http://www.uku.fi/fgts/

MESSAGE FROM THE PRESIDENT Dear Friends, Gene therapy has matured as an exciting new field of research which will have a significant impact on biology and medicine during the next decade. Both pathogenesis of various diseases and basic biological pathways can be studied by gene transfer techniques. Thus, gene transfer will provide a versatile tool for researchers to solve emerging biological questions. However, it is also evident that systematic basic research is needed to improve gene transfer vectors and delivery methods before the full potential of gene transfer techniques can be realized. Finnish scientists have been active in the field of gene therapy for several years. Since the number of researchers engaged in gene transfer and gene therapy studies has steadily increased, it was felt that it is time to establish Finnish Gene Therapy Society. The major aims of the Society are to foster the exchange of ideas between research groups and individual scientists, to organize scientific meetings and training courses and to inform general public about basic aspects and recent developments of gene therapy. Finnish Gene Therapy Society will also establish a Fund for travel grants for promising young scientists. We plan to maintain an updated www home page in order to improve exchange of ideas and information in the field of gene therapy. I am looking forward to working with you to create a very active and stimulating Society. I also welcome all comments and suggestions regarding future activities of the Society.

68. American Scientist: Gene Therapy gene therapy At its core, the notion of gene therapy or gene correctionis scientifically sound. Next Section. © American Scientist 1999. http://www.sigmaxi.org/amsci/articles/99articles/kmiecintro.html

Gene Therapy Eric B. Kmiec Full Text Sections Introduction Gene Addition Adenovirus and Others Targeted Gene Repair Clinical Trials ... May-June 1999 Gene Therapy Investigators have been searching for ways to add corrective genes to cells harboring defective genes. A better strategy might be to correct the defects. Most of the gene therapy techniques developed so far are of the gene-addition variety; that is, they attempt to provide a good copy of a gene to a cell that harbors a bad one. The hope is that the good, corrective gene will compensate for the bad one and restore the cell to its proper function. Gene addition has been achieved by a variety of means not only in test-tube experiments, but in clinical trials involving real patients as well. Yet, to date, the results of these trials have been disappointing. Even the most successful clinical trial has fallen short of therapeutic efficacy. Unfortunately, many of these trials have been widely publicized and, in some cases, oversold in popular books and magazine articles. Having failed to live up to the inflated expectations created by such publicity, these disappointing trial results have left a general impression that gene therapy cannot now or ever fulfill its initial promise. But these clinical trials may have been conducted before the technology was fully mature, driven in part by investor demands on biotechnology companies to rush products to market. Such clinical trials were almost certainly destined to fall short of the mark.

69. Archport Ltd. Home Page Bio-Pharmaceutical Contract Manufacturing Provides contract production and R D services (including animal cell fermentation and downstream processing) to the biopharmaceutical/biotechnology industry, of recombinant proteins. Indepth information on suspension, microcarriers, perfusion, batch fermentation, and gene therapy applications. http://www.archport.ie

70. American Scientist: Gene Therapy MayJune, 1999 Volume 87, No. 3 gene therapy. Investigators have been searchingfor ways to add corrective genes to cells harboring defective genes. http://www.sigmaxi.org/amsci/articles/99articles/Kmiec.html

Suggested Links National Cancer Institute The Institute for Human Gene Therapy: University of Pennsylvania site Gene Browser, sponsored by Nature Technology Corporation. GeneMedNetwork: Resource center for gene related web sites. ... Familial Hypercholesterolemia: Full Text Sections Abstract Introduction Gene Addition Adenovirus and Others Targeted Gene Repair ... Bibliography Illustrations Figure 3 Figure 4 Figure 5 Figure 6 ... May-June, 1999 May-June, 1999 Volume 87, No. 3 Gene Therapy Investigators have been searching for ways to add corrective genes to cells harboring defective genes. A better strategy might be to correct the defects. Keywords Eric B. Kmiec It has been the hope of biomedical researchers to find ways to fix the genes that cause disease. Over a decade ago, when researchers at MIT realized that viruses could be modified to carry corrective genes into cells, gene therapy seemed to be an eventuality. But to date, no one has found a way to reliably control the therapeutic genes to make them clinically useful. Our author surveys the history of this field and gives us a peek at the future of gene therapy, where the emphasis may be less on replacing defective genes and more on correcting them. Sigma Xi About Us Latest Issue Bookshelf ... Web Admin

71. Cancer Gene Therapy Publishes original laboratory and clinical research papers, case reports and review articles covering developments in gene transfer and gene therapy as applied to cancer research. Full articles available by subscription . http://www.naturesj.com/cgt/

nature.com about npg nature science update naturejobs ... help SEARCH my account e-alerts subscribe register ... Journal home For readers Content Online sample issue E-alerts Indexed in ... Society publishing NPG Subject areas Access material from all our publications in your subject area: Biotechnology Cancer Chemistry Clinical Medicine ... Physics ISSN 0929-1903 2003 Volume 10 Publishes 12 issues a year ISI 2001 Impact Factor 2.756 Rank 45/113 Genetics and Heredity, 36/107 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 Official Journal of the International Society of Cancer Gene Therapy Online Submission.....Now available with

72. TKT - Transkaryotic Therapies Develops and commercializes gene activation and nonviral gene therapy products for long term treatment of a broad range of human diseases. (Nasdaq TKTX). http://www.tktx.com/

TKT Announces Reduction in Workforce as Company Focuses on Core Business Areas TKT Provides Update on Participation at Upcoming Health Care Conferences TKT Announces Webcast of Presentation at Upcoming SG Cowen Health Care Conference TKT Announces Reduction in Workforce as Company Focuses on Core Business Areas TKT Provides Update on Participation at Upcoming Health Care Conferences TKT Announces Webcast of Presentation at Upcoming SG Cowen Health Care Conference

73. BBC News | HEALTH | 'Bubble Boy' Saved By Gene Therapy In one of the first treatments of its kind, UK doctors have used gene therapyto cure a toddler of a potentially fatal disorder. gene therapy hurdles. http://news.bbc.co.uk/hi/english/health/newsid_1906000/1906999.stm

CATEGORIES TV RADIO COMMUNICATE ... INDEX SEARCH You are in: Health Front Page World UK ... AudioVideo SERVICES Daily E-mail News Ticker Mobiles/PDAs Feedback ... Low Graphics Wednesday, 3 April, 2002, 12:24 GMT 13:24 UK 'Bubble boy' saved by gene therapy Rhys can now play outside In one of the first treatments of its kind, UK doctors have used gene therapy to cure a toddler of a potentially fatal disorder. Scientists at Great Ormond Street Hospital in London successfully treated 18-month-old Rhys Evans, who had a condition preventing him from developing an immune system. The problem, called severe combined immunodeficiency (SCID) is caused by a single mutated gene, and meant that he had to live in sterile conditions or risk picking up a life-threatening infection. After his gene therapy, he was running around at home - he's a normal little boy now Dr Adrian Thrasher, Great Ormond Street Hospital Rhys, from Treharris, nr Cardiff, had spent much of his life in hospital prior to the treatment. Only one in between 50,000 and 100,000 births in the UK is affected by the disorder.

74. Cystic Fibrosis Clinical And Research Center - Children's Hospital And Regional University of Washington CF Center. Information on Cf Research Development Program, Core Center for gene therapy, Clinic, Research Trials, current events and staff. http://depts.washington.edu/cfcenter

Core Center for Gene Therapy CF Research Development Program (CF RDP Cystic Fibrosis Clinics Clinical Research Trials Therapeutic Development Center Request for Applications Pilot and Feasibility Projects Investigator's Seminar Series 2001-2002 schedule Faculty and Staff ... Contact info Funding Opportunities Bulletin Board Children's Hospital and Regional Medical Center, Seattle WA Last update 5/17/01 Website created and designed by Khanh Vu. Contact info: kv113@u.washington.edu

75. University Of Texas M. D. Anderson Cancer Center Gene Therapy Department The human cancer gene prevention and therapy program conducts basic research, clinical trials and Category Science Biology Animal Mammal Human gene therapy......Welcome to the WM Keck Center for Cancer gene therapy website. Genetherapy is the transfer of normal or redesigned genes into a http://www.mdanderson.org/depts/genetherapy/

Message from the Program Directors Program Members Institutional Vector Core What's New ... Gene Therapy Links Development Awards Welcome to the W.M. Keck Center for Cancer Gene Therapy website. Gene therapy is the transfer of normal or redesigned genes into a patient to reduce or alleviate a disease state. Certain potentially cancerous genes are dormant in all of us and are sometimes triggered into activity. Other genes, tumor-suppressor genes, act naturally to stop unregulated growth of cancer cells. Please browse this site to learn more about the Keck Center. Message from the Program Directors The gene therapy program at the University of Texas M.D. Anderson Cancer Center has assembled over 50 scientists and clinicians with an interest in sharing research ideas and findings worldwide. We all share the common goal of curing cancer Membership If you are Interested in joining the W.M. Keck Center for Cancer Therapy, please forward Debbie Waits a current copy of your CV and your research interests in gene therapy. Ms. Waits' e-mail address is dwaits@mail.mdanderson.org.

76. MEDLINEplus: Genes And Gene Therapy Search MEDLINE for recent research articles on Genes and gene therapy •gene therapy • Gene mapping. More News on Genes and gene therapy. http://www.nlm.nih.gov/medlineplus/genesandgenetherapy.html

Skip navigation Other health topics: A B C D ... List of All Topics Genes and Gene Therapy Contents of this page: News From the NIH General/Overviews Anatomy/Physiology ... Teenagers Search MEDLINE for recent research articles on Genes and Gene Therapy: Gene therapy Gene mapping You may also be interested in these MEDLINEplus related pages: Genetic Disorders Genetic Testing/Counseling Genetics/Birth Defects Latest News Two Genes Found Key in Parkinson's Risk in Women (03/20/2003, Reuters Health) Obesity Found to Have Many, Varied Causes (03/20/2003, United Press International) Some Cases of Obesity Caused by a Single Gene (03/19/2003, Reuters Health) FDA Advisory Committee Discusses Steps for Potentially Continuing Certain Gene Therapy Trials That Were Recently Placed on Hold (02/28/2003, Food and Drug Administration) FDA Places Temporary Halt on Gene Therapy Trials Using Retroviral Vectors in Blood Stem Cells (01/14/2003, Food and Drug Administration) More News on Genes and Gene Therapy From the National Institutes of Health Frequently Asked Questions About Genetics (National Human Genome Research Institute) Human Genome Project (National Human Genome Research Institute) Questions and Answers About Gene Therapy (National Cancer Institute) General/Overviews Gene Therapy (Dept. of Energy)

77. Another Chance For Gene Therapy? An 18year-old man has died after undergoing experimental gene therapy for a rare metabolic disease. Researchers say it shouldn't stigmatize all gene therapy. By Kristen Philipkoski. Wired News http://www.wired.com/news/news/story/22036.html

78. All The Virology On The WWW - Viral Vectors And Gene Therapy An up to date index of gene therapy and Viral Vector site, from All the Virologyon the WWW. Please visit our sponsor! Viral Vectors and gene therapy. http://www.virology.net/garryfavwebgenether.html

Please visit our sponsor! Viral Vectors and Gene Therapy: General Sites Gene Therapy Related Sites Clinical Trials E-Journals Conferences Related Resources: Scientific Meetings Scientific Journals ATV Home ATV Table of Contents ... Graffiti Wall David M. Sander, Ph.D. (david.sander@virology.net ) If you are interested in developing a WWW site for your lab or organization, please feel free to contact me for any needed advice and/or assistance. Viral Vectors and Gene Therapy General Sites Report and Recommendations of the Panel to Assess the NIH Investment in Research on Gene Therapy - Dr. Harold Varmus, Director, National Institutes of Health (NIH), appointed an ad hoc committee to assess the current status and promise of gene therapy and provide recommendations regarding future NIH-sponsored research in this area. The Panel was asked specifically to comment on how funds and efforts should be distributed among various research areas and whatfunding mechanisms would be most effective in meeting research goals. Biotechnology and Gene Therapy Web 'Cite' - a large index of Gene Therapy information on the net.

79. Vanderbilt Radiation Oncology Research at a hightechnology treatment facility clinical trials; biological modifiers; therapeutic targets; industry validation of efficacy of pharmaceutical, imaging and radiotherapy methods; medical physics of radiotherapy delivery; radiation-guided drug delivery and gene therapy imaging. Regional facilities. http://www.mc.vanderbilt.edu/radiationoncology

Biological Sciences Medical Physics ... Education Mission Statement The mission of the Department of Radiation Oncology is to improve the treatment outcomes of all diseases treated with radiation and to find new medical uses of radiation. Related to these goals are the education of graduate students, post-doctoral students and residents who will enter the field of radiation medicine including the allied fields of Medical Physics and Radiation Therapy. The Department of Radiation Oncology provides state-of-the-art anti-cancer therapy for patients through the use of new technology and through the continuous review of patient treatment outcome. We will continue to evaluate and improve the therapeutic effects of radiation from five fronts: 1) Basic science research to indentify new biological modifiers and therapeutic targets. 2) Interaction with industry (pharmaceutical, imaging and radiotherapy) to validate the efficacy of new therapeutic tools. 3) Medical phyiscs research to improve the accuracy, precision and methods of radiotherapy delivery. 4) Clinical research to study the benefits of new therapy treatments in patients 5) Development of new radiotherapy facilities throughout the region to provide the best care for all patients.

80. The Program In Biomedical Sciences (PIBS) - Gene Therapy gene therapy. Yehoash Raphael, Ph.D. Design of biologically modified cochlear implantsfor combining electrical stimulation with gene therapy in the inner ear. http://www.med.umich.edu/pibs/faculty/gene_therapy.htm

Gene Therapy Please note: Words in italics are the official PIBS affiliations of the indicated faculty. James R. Baker Jr., MD Determining why autoantigens are recognized in autoimmune disease; epitope mapping thyroid autoantigens from patients with autoimmune thyroid disease. Immunology. Pathology. Kate F. Barald, Ph.D. Developmental neurobiology; molecular neurobiology studies of developing neuronal cells and synapses; gene expression in the neural crest; molecular studies of inner ear development. Cell and Developmental Biology. Neuroscience. Cellular and Molecular Biology. D. Keith Bishop, Ph.D. Cellular immunology of transplant rejection and acceptance. Microbiology and Immunology. Immunology. Sally A. Camper, Ph.D. Developmental genetics of the neuroendocrine system; transgenic mice; gene targeting; pituitary dwarfism. Human Genetics. Bioinformatics. Cellular and Molecular Biology. Neuroscience. Michael F. Clarke, MD

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