First evidence of gene therapy for abnormal blood vessel growth in newbornsJuly 18, 2001The first evidence of the potential for gene therapy to treat eye disease that stems from abnormal blood vessel growth is revealed in research published in the British Journal of Ophthalmology. Gene therapy is a relatively new and much heralded therapeutic approach. But despite the advances in molecular genetics, attempts to target organs or particular body structures using the technique have proved disappointing. The problem appears to hinge on the vector agent that is used to target cellular DNA. It must be capable of successfully delivering a genetic sequence and at the same time be able to withstand the receiving body's immune system response. The researchers studied newborn rats with induced retinopathy of prematurity. This is a disease which is on the increase as a result of the rising numbers of surviving premature babies. It is a leading cause of blindness and defective vision in children, and despite surgical advances, around half of babies with the most severe form of the disease will become blind. Disordered blood vessel growth from the retina needs to be prevented in order to treat the disease. The researchers tested the ability of four different viruses, adenovirus, bovine smallpox virus, herpes virus, and a virus that causes leukaemia, the murine leukaemia virus, to transport genetic material into the defective eyes of 18 day old rats. The results showed that adenovirus was the most effective vector, and furthermore it specifically targeted tissues in the jelly-like material inside the eyeball which destroy vital developing eye tissues. Delicate adjacent nerve tissues risk being damaged if the target is not specific. The research is in its early stages, say the authors, but they suggest that this is a first step in the use of gene therapy for both treatment and research purposes. British Medical Journal (BMJ) |
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| Related Gene Therapy Current Events and Gene Therapy News Articles Research 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. More Gene Therapy Current Events and Gene Therapy News Articles |
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