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Gene therapy breakthrough offers hope to patients with inherited high cholesterol levels
May 14, 2002New research published in BMC Molecular Biology explains how a new technique for introducing genes into mammalian cells using the virus responsible for warts could be a major step forward in developing gene therapy treatments for people with familial hypercholesterolemia (FH), a genetic disease that affects around 12 million people worldwide.
People with FH have a genetic defect that prevents their liver cells from absorbing chlolesterol in the form of low density lipoprotein (LDL). This leads to high levels of cholesterol in the bloodstream increasing the risk of heart disease. It is estimated that about half of men and a third of women with FH suffer a heart attack by the age of 60.
FH occurs as a result of a single defective gene that codes for a LDL receptor in the liver. Many single gene defects like the one that causes FH are candidates for gene therapy, a medical treatment used to repair or replace faulty genes (this is because it is much easier to repair one gene that causes a disease than it is to tackle other genetic diseases that involves several faulty genes).
Since the first successful attempts at gene therapy in 1990 there has been an intense research effort to develop ways of curing genetic diseases. By 1996 over 3000 patients had been treated using gene therapy, but most of these cases were unsuccessful. There are major obstacles to overcome to increase the success of gene therapy trials. Firstly, ways of getting multiple copies of healthy genes into enough cells to reverse the underlying disease need to be established. Secondly, it is important that the expression of healthy genes is sustained and finally any virus used to repair or replace faulty genes must not be attacked by the immune system of the patient receiving gene therapy.
The research published in BMC Molecular Biology showed that the virus responsible for warts (bovine papilloma virus) could be used to restore the function of cells with defective LDL receptors. Furthermore, it showed that the cells continued to function normally throughout the 32 week observation period.
The next step in this exciting research is to test the ability of the virus to repair faulty genes in animals with defective LDL receptor genes to establish whether an animal`s immune system will react to the papilloma virus. The researchers remain optimistic that this technique will save lives given that preliminary studies have shown that the human form of the virus is unlikely to attacked by the immune system.
The full text of this article is freely available in the online peer-reviewed journal BMC Molecular Biology http://www.biomedcentral.com/1471-2199/3/5/
BioMed Central Limited
Since the first successful attempts at gene therapy in 1990 there has been an intense research effort to develop ways of curing genetic diseases. By 1996 over 3000 patients had been treated using gene therapy, but most of these cases were unsuccessful. There are major obstacles to overcome to increase the success of gene therapy trials. Firstly, ways of getting multiple copies of healthy genes into enough cells to reverse the underlying disease need to be established. Secondly, it is important that the expression of healthy genes is sustained and finally any virus used to repair or replace faulty genes must not be attacked by the immune system of the patient receiving gene therapy.
The research published in BMC Molecular Biology showed that the virus responsible for warts (bovine papilloma virus) could be used to restore the function of cells with defective LDL receptors. Furthermore, it showed that the cells continued to function normally throughout the 32 week observation period.
The next step in this exciting research is to test the ability of the virus to repair faulty genes in animals with defective LDL receptor genes to establish whether an animal`s immune system will react to the papilloma virus. The researchers remain optimistic that this technique will save lives given that preliminary studies have shown that the human form of the virus is unlikely to attacked by the immune system.
The full text of this article is freely available in the online peer-reviewed journal BMC Molecular Biology http://www.biomedcentral.com/1471-2199/3/5/
BioMed Central Limited
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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