Extractions: 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. 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. Levels greater than 20% to 30% would be sufficient to prevent bleeding in all but the most traumatic injuries.
Gene Therapy Links Genetix Links on gene therapy. Applying Genomics to Disease and Therapy The Bystander Effect In gene therapy Great, But How Does It Work? http://www.genetixpharm.com/genes.htm
Applied Tissue Technologies - Home Licenses advanced core technologies for wound healing and gene therapy. Based in Wellesley Hills, MA. http://www.appliedtissue.com/
Extractions: home gene therapy stem cells microseeding patents ... contact We deliver genes to skin and bone using naked DNA or any chemical or viral vector. Our method for transplanting genetically engineered cells is based on two components: Our Microseeding technology transfers genetic material directly into an external or internal target using a microneedle delivery device to deliver the material through repeated punctures of the target area.
UK Cystic Fibrosis Gene Therapy 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. http://www.cfgenetherapy.org.uk/
Extractions: Home Members Research Publications ... 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 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. We would be interested to hear from any researchers who would like to test a gene transfer agent in these core facilities. For more information contact products@cfgenetherapy.org.uk
FINNISH GENE THERAPY SOCIETY - MAIN PAGE Fosters the exchange of ideas between research groups and individual scientists, organizes scientific meetings and training courses and informs general public about basic aspects and recent developments in gene therapy. http://www.uku.fi/english/organizations/fgts/index.htm
Extractions: 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.
Washingtonpost.com Jesse Gelsinger gene therapy Death Jesse Gelsinger, 18, died in September 1999 after a failed experimental therapy administered by the University of http://www.washingtonpost.com/wp-dyn/health/specials/genetherapy/
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PNP Therapeutics, Inc. Company developing gene therapy system, using the E. coli enzyme, purine nucleoside phosphorylase, to induce suicide in tumour cells. Includes technology background, research, patents, publications, key staff, and contacts in Birmingham, Alabama. http://www.pnptherapeutics.com/
Extractions: PNP Therapeutics , Inc. is an emerging biotechnology company engaged in the research and development of a platform technology and proprietary products for the treatment of cancer. PNP Therapeutics , Inc. was founded to fully exploit the cancer curing potential of the E.coli enzyme, purine nucleoside phosphorylase (PNP). The Company is dedicated to the successful development and commercialization of a unique suicide gene therapy approach that is predicated on the knowledge that solid tumors can be compelled to develop their own chemotherapy and thus elicit their own self-destruction. Many common cancers (including prostate, breast, colon, lung, brain, melanoma, pancreas, ovarian, kidney) can progress to become untreatable and eventually kill the patient. Compounds are available that could abolish these tumors, but are too toxic to administer safely to cancer patients. We have shown that some of these compounds are remarkably potent and can abolish otherwise refractory human cancers when produced within the tumor mass. 4363 1st Avenue North, Birmingham, Alabama 35222 USA
Virus Vectors & Gene Therapy Virus Vectors and gene therapy A review by David Peel, University of Leicester. Virus Vectors gene therapy Problems, Promises Prospects. David Peel. http://www.tulane.edu/~dmsander/WWW/335/peel/peel1.html
Extractions: 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. ^TOP^ Next Section University of Leicester
The Journal Of Gene Medicine Articles on gene therapy including design and production of vectors, research into the mechanisms underlying gene transfer, preclinical studies including animal models, developmental aspects and clinical trials. http://www.interscience.wiley.com/jpages/1099-498X/
Wired News: Gene Therapy Shows Its Muscle gene therapy Shows Its Muscle. Blood and urine tests can detect most performanceenhancing drugs, but the gene therapy detection would be much more difficult. http://www.wired.com/news/medtech/0,1286,62316,00.html
Extractions: 06:59 PM Feb. 16, 2004 PT SEATTLE A gene therapy that has been shown in rats to double muscle strength and power could illegally be used to build super athletes, a researcher said Monday. Sports officials are looking for ways to detect the genetic manipulation. Lee Sweeney of the University of Pennsylvania said laboratory studies show that injecting a virus carrying the gene for insulin-like growth factor 1 into lab rats caused their target muscles to grow in size and strength by 15 to 30 percent. Report: Lab Results Counter Bryant Accuser's Story Dominican, Haiti Floods Death Toll Nears 2,000
Wired News: Feds Grapple With Gene Therapy Feds Grapple With gene therapy. A government agency says gene therapy experiments should be allowed to continue, despite pleas for a moratorium on the research. http://www.wired.com/news/technology/0,1282,34766,00.html
Extractions: 03:00 AM Mar. 14, 2000 PT A government agency says gene therapy experiments should be allowed to continue, despite pleas for a moratorium on the research. The Recombinant DNA Advisory Committee met from 8 March through 10 March in Washington to discuss ways to better safeguard patients during gene therapy experiments. Wireless Hot Spot Directory Search for Wi-Fi hot spots near you: Tech Jobs Partner Today's the Day The 15-member committee, while unable to come to terms on exactly how gene therapy experiments would continue, did agree they should be allowed, in spite of public concern and a death last year due to experimental gene therapy.
Gene Therapy - Wikipedia, The Free Encyclopedia gene therapy. From Wikipedia, the free encyclopedia. gene therapy is the insertion the genome. Types of gene therapy. In theory it http://en.wikipedia.org/wiki/Gene_therapy
Extractions: Other languages: Dansk Deutsch Nederlands 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 6 External links 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
Human Gene Therapy A reference resource for the latest information concerning human gene therapy. http://www.humangenetherapy.com/
Extractions: Select Subscription Rate: -Personal Rates- 2004 Online Only Personal $699.00 2004 Outside USA Print Personal $1,075.00 2004 USA Print Personal $746.00 -Institutional Rates- 2004 Online Only Institutional $1,899.00 2004 Outside USA Print Institutional $2,245.00 2004 USA Print and Online Institutional $2,379.00 2004 USA Print Institutional $1,988.00 Please note subscription orders are entered for the complete volume year. For example, if you are ordering the 2004 volume, you will receive any and all issues published to date for that volume. HUMAN GENE THERAPY LAUNCHES ELECTRONIC MANUSCRIPT SUBMISSION AND PEER REVIEW EFFECTIVE MAY 24, 2004
Wiley - The Journal Of Gene Medicine Website An information source on all aspects of human gene therapy. It offers an international comprehensive summary of both published and ongoing clinical trials. Also includes reviews on basic and therapeutic issues. http://www.wiley.co.uk/genetherapy/
Don Hill And Associates Offer handson experience in interpreting and applying cGMPs and regulatory requirements to the fields of pharmaceutical products, vaccines, cellular and gene therapy and traditional small molecule drugs. Contacts by email or phoning Maryland. http://www.donhillassociates.com
Extractions: The company is comprised of four former FDA officials and two former pharmaceutical industry associates, all with hands-on experience in interpreting and applying cGMPs and regulatory requirements. Our clients include major multi-national pharmaceutical companies, small venture-capital start up operations, and virtual companies. Don Hill and his select cadre of qualified associates bring many years of FDA regulatory experience or pharmaceutical industry experience to help you achieve your objectives and needs in the fields of biotechnology products, vaccines, cellular and gene therapy and more recently in traditional small molecule drugs. This experience includes: Performing cGMP and cGLP audits of facilities, manufacturing, operations and documentation; preparing clients for pre-approval, biennial and "for cause" cGMP inspections;
Gene Therapy Sites gene therapy An Overview From Biotech Applied gene therapy Potential, Pitfalls and Prospects By Inder M. Verma for an NIH Director s Wednesday Afternoon http://www.uq.edu.au/vdu/GeneTherapylinks.htm
Gene Therapy Home Page Publishes research about the identification of gene structure, disease processes and the development of clinical methods to treat disease. Includes instructions for authors and related journals. http://www.naturesj.com/gt/
Extractions: 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 36/266 Biochemistry and Molecular Biology View tables of contents At the forefront of medicine, Gene Therapy brings you the latest research into genetic and cell-based technologies and the development of clinical methods to treat disease. online submission..... Now available with Gene Therapy Advance online publication (AOP)
The Scientist - Gene Therapy's Gold Rush News. gene therapy s Gold Rush. By Kathryn S. Brown. Author Kathryn S. Brown. Sidebar Major Pharmaceutical Companies Are Diving Into the gene therapy Pool. http://www.the-scientist.com/yr1995/nov/capital_951113.html
Extractions: News By Kathryn S. Brown Into Gene Therapy Research Cautious observers note, however, that the fate of the new industry may hinge on a flurry of recently approved trials. Author: Kathryn S. Brown Sidebar Major Pharmaceutical Companies Are Diving Into the Gene Therapy Pool As gene therapy enters its sixth year of high-visibility research, major drug companies are buying in, helping lay the technology's foundation and launching clinical trials. More than 100 gene therapy trials-most privately funded-are under way. And while the field's ultimate success is still quite uncertain, one thing is clear: There is a lot of science to be done and research opportunities to be pursued. ACADEMIA, INDUSTRY UNITE : Jack Roth of U. Texas M.D. Anderson Cancer Center lauds drug firms' investment. "It seems like there is a new company announcing a gene therapy project every week," says James Barrett, chief executive officer of Genetic Therapy Inc. (GTI), a publicly held biotechnology company in Gaithersburg, Md. "There is a whole wealth of opportunity for scientists." BUSTLE : James Barrett notes that gene therapy provides "a whole wealth of opportunity for scientists."
Dept. Of Molecular Microbiology & Immunology Ph.D. degree training focusing on hostpathogen interactions, include virology, viral pathology, biodefense, cancer gene therapy, and vaccine development. Site includes information on curriculum and faculty research. http://medschool.slu.edu/mmi