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Moving gene therapy forward with mobile DNA
May 04, 2009Gene therapy
Gene therapy is the introduction of genetic material into a patient's cells resulting in a cure or a therapeutic effect. In recent years, it has been shown that gene therapy is a promising technology to treat or even cure several fatal diseases for which there is no attractive alternative therapy. Gene therapy can be used for hereditary diseases, but also for other diseases that affect heart, brain and even for cancer. Indeed, recent results suggest that gene therapy can be beneficial for patients suffering from aggressive brain cancer that would otherwise be lethal.
A safe delivery of the genes?
Despite the overall progress, there is still a need to develop improved and safer approaches to deliver genes into cells. The success of gene therapy ultimately depends on these gene delivery vehicles or vectors. Most vectors have been derived from virusses that can be tailor-made to deliver therapeutic genes into the patients' cells. However, some of these viral vectors can induce side-effects, including cancer and inflammation.
Marinee Chuah, Thierry VandenDriessche, Eyayu Belay and their fellow VIB researchers at K.U. Leuven in collaboration with Zsuzsanna Iszvak and Zoltan Ivics and colleagues at the Max Delbrück Center in Berlin (Germany) have now developed a new non-viral approach that overcomes some of the limitations associated with viral vectors.
Lessons from evolution
Using the principles of evolution and natural selection, that were initially conceived by Charles Darwin, they have now developped an efficient and safe gene delivery approach based on non-viral genetic elements, called transposons. Transposons are mobile DNA elements that can integrate into 'foreign' DNA via a 'cut-and-paste' mechanism. In a way they are natural gene delivery vehicles. The researchers constructed the transposons in such a way that they can carry the therapeutic gene into the target cell DNA. Doing so, they obviate the need to rely on viral vectors.
'We show for the first time that it is now possible to efficiently deliver genes into stem cells, particularly those of the immune system, using non-viral gene delivery', says Marinee Chuah. 'Many groups have tried this for many years but without success. We are glad that we could now overcome this hurdle' claims Thierry VandenDriessche. Zsuzsanna Izsvak and Zoltan Ivics concur: 'This transposon technology may greatly simplify the way gene therapy is conducted, improve its overall safety and reduce the costs'.
The VIB researchers are further testing this technology to treat specific diseases including cancer and genetic disorders, in anticipation of moving forward and treat patients suffering from these diseases.
VIB (the Flanders Institute for Biotechnology)
Despite the overall progress, there is still a need to develop improved and safer approaches to deliver genes into cells. The success of gene therapy ultimately depends on these gene delivery vehicles or vectors. Most vectors have been derived from virusses that can be tailor-made to deliver therapeutic genes into the patients' cells. However, some of these viral vectors can induce side-effects, including cancer and inflammation.
Marinee Chuah, Thierry VandenDriessche, Eyayu Belay and their fellow VIB researchers at K.U. Leuven in collaboration with Zsuzsanna Iszvak and Zoltan Ivics and colleagues at the Max Delbrück Center in Berlin (Germany) have now developed a new non-viral approach that overcomes some of the limitations associated with viral vectors.
Lessons from evolution
Using the principles of evolution and natural selection, that were initially conceived by Charles Darwin, they have now developped an efficient and safe gene delivery approach based on non-viral genetic elements, called transposons. Transposons are mobile DNA elements that can integrate into 'foreign' DNA via a 'cut-and-paste' mechanism. In a way they are natural gene delivery vehicles. The researchers constructed the transposons in such a way that they can carry the therapeutic gene into the target cell DNA. Doing so, they obviate the need to rely on viral vectors.
'We show for the first time that it is now possible to efficiently deliver genes into stem cells, particularly those of the immune system, using non-viral gene delivery', says Marinee Chuah. 'Many groups have tried this for many years but without success. We are glad that we could now overcome this hurdle' claims Thierry VandenDriessche. Zsuzsanna Izsvak and Zoltan Ivics concur: 'This transposon technology may greatly simplify the way gene therapy is conducted, improve its overall safety and reduce the costs'.
The VIB researchers are further testing this technology to treat specific diseases including cancer and genetic disorders, in anticipation of moving forward and treat patients suffering from these diseases.
VIB (the Flanders Institute for Biotechnology)
Gene Therapy Current Events and Gene Therapy News RSSResearch reveals lipids' unexpected role in triggering death of brain cells
The lipid that accumulates in brain cells of individuals with an inherited enzyme disorder also drives the cell death that is a hallmark of the disease, according to new research led by St. Jude Children's Research Hospital investigators.
No-entry zones for AIDS virus
The AIDS virus inserts its genetic material into the genome of the infected cell. Scientists of the German Cancer Research Center have now shown for the first time that the virus almost entirely spares particular sites in the human genetic material in this process. This finding may be useful for developing new, specific AIDS drugs.
Cornell researchers identify a weak link in cancer cell armor
The seeming invincibility of cancerous tumors may be crumbling, thanks to a promising new gene therapy that eliminates the ability of certain cells to repair themselves.
Treatment to improve degenerating muscle gains strength
A study appearing in Science Translational Medicine puts scientists one step closer to clinical trials to test a gene delivery strategy to improve muscle mass and function in patients with certain degenerative muscle disorders.
Iowa State University researcher discovers key to vital DNA, protein interaction
A researcher at Iowa State University has discovered how a group of proteins from plant pathogenic bacteria interact with DNA in the plant cell, opening up the possibility for what the scientist calls a "cascade of advances."
Scientists successfully reprogram blood cells
Researchers have transplanted genetically modified hematopoietic stem cells into mice so that their developing red blood cells produce a critical lysosomal enzyme -preventing or reducing organ and central nervous system damage from the often-fatal genetic disorder Hurler's syndrome.
Immune therapy can protect against or treat later lymphoma
Specially developed immune system cells that target the common Epstein-Barr virus can protect immune-suppressed bone marrow transplant recipients against lymph system disease and cancers that arise from the viral infection.
Caltech researchers show efficacy of gene therapy in mouse models of Huntington's disease
Researchers at the California Institute of Technology (Caltech) have shown that a highly specific intrabody (an antibody fragment that works against a target inside a cell) is capable of stalling the development of Huntington's disease in a variety of mouse models.
Immunotherapy demonstrates long-term success in treating lymphoma
Targeted immunotherapy has been an attractive new therapeutic area for a number of cancers because it has the potential to destroy tumor cells without damaging surrounding normal tissue. New study results demonstrate high success rates using specialized white blood cells to prevent or treat lymphoma associated with the Epstein-Barr virus (EBV-lymphoma) in patients who have received a hematopoietic stem cell transplant (HSCT).
Toward bold new anti-cancer medicines
Bold new strategies in the battle against cancer may turn forms of the disease that presently are incurable into manageable conditions that can be controlled for long periods of time.
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Gene Therapy: Treating Disease by Repairing Genes (New Biology) by Joseph, Ph.D. Panno (Author) Many diseases are caused by a simple point mutation - they are the result of a single defective gene. Gene therapy is the attempt to replace a defective gene with a good copy or to add a good gene to compensate for the bad. Although this procedure has proven to be extremely hazardous, of all the technologies provided by the new biology, gene therapy holds much potential for curing disease. Dealing with one of the most controversial aspects of the new biology, Gene Therapy explores the development and future of this area of research and explains why it is considered such a promising branch of science. This encyclopedia presents an overview of genetic defects, viruses, and gene therapy in a way that students and general readers will find easy to understand. Comprehensive in scope, this book... |
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Gene and Cell Therapy: Therapeutic Mechanisms and Strategies, Third Edition by Nancy Smyth Templeton (Editor) Since the publication of the second edition of this book in 2004, gene therapy and cell therapy clinical trials have yielded some remarkable successes and some disappointing failures. Now in its third edition, Gene and Cell Therapy: Therapeutic Mechanisms and Strategies assembles many of the new technical advances in gene delivery, clinical applications, and new approaches to the regulation and modification of gene expression. New Topics Covered in this Edition: Gene and Cell Therapies for Diabetes and Cardiovascular Diseases Clinical Trials Human Embryonic Stem Cells Tissue Engineering Combined with Cell Therapies Novel Polymers Relevant Nanotechnologies SiRNA Therapeutic Strategies Dendrimer Technologies Comprised of... |
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Gene Therapy and Gene Delivery Systems (Advances in Biochemical Engineering / Biotechnology) by David V. Schaffer (Editor), Weichang Zhou (Editor) 1 D.V. Schaffer, W. Zhou: Gene Therapy as Future Human Therapeutics.- 2 J. Heidel, S. Mishra, M.E. Davis: Molecular Conjugates.- 3 M. Manthorpe, P. Hobart, G. Hermanson, M. Ferrari, A. Geall, B. Goff, A. Rolland: Plasmid Vaccines and Therapeutics: From Design to Applications.- 4 S.R. Little, R. Langer: Non-Viral Delivery of Cancer Genetic Vaccines.- 5 J.C. Grieger, R.J. Samulski: Adeno-Associated Virus as a Gene Therapy Vector: Vector Development, Production and Clinical Applications.- 6 J.H. Yu, D.V. Schaffer: Advanced Targeting Strategies for Murine Retroviral and Adeno-Associated Viral Vectors.- 7 N. Loewen, E.M. Poeschla: Lentiviral Vectors.- 8 N.E. Altaras, J.G. Aunins, R.K. Evans, A. Kamen, J.O. Konz, J.J. Wolf: Production and Formulation of Adenovirus Vectors.- |
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Gene Therapy & Molecular Biology by Intl Soc Gene Therapy Mole Bio | |
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Gene Therapy by Mary Colavito (Author), Michael A. Palladino (Author) KEY MESSSAGE: Will gene therapy one day become the ultimate application of genetic technology? Gene Therapy, the newest booklet in the Special Topics in Biology Series, addresses this question by exploring gene therapy as a complex technology for delivering therapeutic genes as a way to treat and cure human genetic diseases. Author Mary Colavito provides an overview of the basic science involved in gene therapy methods and chronicles the history of gene therapy by discussing successful and ongoing gene therapy treatments as well as adverse outcomes in some cases of gene therapy. Also discussed are challenges that must be overcome for gene therapy to become a more reliable and readily accessible approach for treating a multitude of genetic diseases that affect... |
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Jazz Therapy, Vol. 1: Smile by Gene Bertoncini & Roni Ben-Hur The first in a new series of fundraising CDs produced in association with the Jazz Foundation of America, and the first time these two popular guitarists have recorded together. Liner notes by Ira Gitler. Entire CD was played, recorded, packaged, and is being promoted on a pro-bono basis, raising funds for the Englewood Hospital and Medical Center's Dizzy Gillespie Fund. |
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Gene Therapy: Dawn of a New Age in Medicine (NCME Video 653) Also With: Kenneth W. Culver (Primary Contributor), Network For Continuing Medical Education (Primary Contributor) |
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Six Years of Gene Therapy Son of Rust (Primary Contributor) |
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Chromosome 11 Gene Therapy Sickle Cell Anemia Photographic Poster Print, 24x32 by Art.com Art.com is the world's largest retailer of art prints, posters, photographs, and framed artwork. With our huge selection of over 400,000 prints, you'll easily find the perfect piece for your home, office, or classroom. Our art is printed on quality paper. When you order framed artwork, the piece is built by our team of in-house professionals. Visit our Amazon store today at www.amazon.com/artdotcom to find Special Offers and search for products based on 'Artist Name' and 'Subject Categories' such as Movie, Music, Vintage, TV, Children, Travel, Kitchen, Museum Art, Animals, Floral, Motivational, and Sports. Art.com is dedicated to providing you with high quality products and service by offering you 100% satisfaction guaranteed. We ship internationally to over 80 countries. Decorate your... |
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Charlie Rose (August 30, 2000) A rebroadcast of an interview that originally aired on June 21, 2000. Part three of the special edition series, "Mapping the Human Genome." First, three prominent scientists on how the decoding of the genome will lead to breakthroughs in the battle against disease. They are Dr. Harold Varmus, president of Memorial Sloan-Kettering Cancer Center, Dr. Arnold Levine, president of Rockefeller University, and Dr. Savio Woo, president of the American Society of Gene Therapy.||Then, Dr. James Watson, Nobel laureate, co-discoverer of the double-helix structure of DNA and president of Cold Springs Harbor Laboratory, discusses public and private genome projects and his new book, a collection of his essays over the years called A Passion for DNA. This product is manufactured on demand using DVD-R... |
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