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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 RSSImmune 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.
1 shot of gene therapy and children with congenital blindness can now see
Born with a retinal disease that made him legally blind, and would eventually leave him totally sightless, the nine-year-old boy used to sit in the back of the classroom, relying on the large print on an electronic screen and assisted by teacher aides.
Master regulator found for regenerating nerve fibers in live animals
Researchers at Children's Hospital Boston report that an enzyme known as Mst3b, previously identified in their lab, is essential for regenerating damaged axons (nerve fibers) in a live animal model, in both the peripheral and central nervous systems.
Governor recognizes stem cell research at Einstein
Albert Einstein College of Medicine of Yeshiva University hosted a roundtable discussion on stem cell research with New York Governor David A. Paterson today.
Michigan hospital launches gene therapy study for Parkinson's disease
A Michigan hospital is embarking on a research study for advanced Parkinson's disease using a state-of-the-art treatment called gene transfer.
Historic gene therapy trial to treat Alzheimer's disease underway at Georgetown
Researchers in the Memory Disorders Program at Georgetown University Medical Center are now recruiting volunteers for a national gene therapy trial - the first study of its kind for the treatment of patients with dementia due to Alzheimer's disease.
University of Iowa scientists use blood-brain barrier as therapy delivery system
The blood brain barrier is generally considered an obstacle to delivering therapies from the bloodstream to the brain. However, University of Iowa researchers have discovered a way to turn the blood vessels surrounding brain cells into a production and delivery system for getting therapeutic molecules directly into brain cells.
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Immunology of Gene Therapy by Roland W. Herzog (Author) Summarises and reviews the important field of genetic therapy with respect to the latest immunological advances in the lab and clinic Unique treatment of immunology and immunotherapy in gene - approached from a vector and target organ point of view rather than from the perspective a specific diseases Broad appeal - applicable for immunology and genetics / gene therapy, recombinant DNA studies, transplantation, virology, cancer research and tumor research |
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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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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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