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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 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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