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Friday, 05 December 2014 11:18

Researchers revealed the way for success in gene therapy

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Researchers-revealed-the-way-for-success-in-gene-therapyScientists have found a new way to overcome one of the biggest obstacles to the use of viruses for therapeutic genes.
Scientists from the Institute for research at national level have found a way to overcome one of the biggest obstacles to the use of viruses for administration of therapeutic genes, that is, how to prevent the immune system to neutralize the virus before it has delivered its genetic set.
Gene therapy is one of the most promising possibilities for the treatment of genetic disorders such as muscular dystrophy, congenital blindness and hemophilia. Scientists explore gene therapy as a cure for certain types of cancer, neurodegenerative diseases, viral infections and other acquired diseases. In order to obtain a therapeutic gene into cells, scientists use viruses that deliver its genetic material in cells as part of their normal replication process.
Again and again, these efforts were thwarted by the very immune system of the body that damages each viral vector. Thus the therapeutic gene can be delivered to diseased cells and disease raging in full force.
A team led by Louis-Rodino Klapak, PhD, and Jerry Mendel, MD, principal investigator in the Center for Gene Therapy at Nationwide, show for the first time that using a process called plasmapheresis, just before delivery of the virus for gene therapy he is protected long enough to enter the cell and deliver their genetic material.
In a study of gene therapy for the treatment of Duchenne muscular dystrophy (DMD), Dr. Rodino-Klapak using plasmapheresis in a large animal model, and then injected a virus carrying the gene micro-dystrophin. When studying the level of gene expression of the micro-dystrophin in animals, it was found that there is a 500% increase in the gene expression in the animals who received a plasmapheresis.
Dr. Mendel believes that right now, gene therapy seems to work best in patients who have antibodies to the virus. It is this virus is used to supply the necessary gene. It is exactly this gene, which is a therapeutic, curative intent of the organism suffering from a disease. On the other hand, it limits the number of patients who can benefit from gene therapy. This is because in very few patients lacking antibodies against the virus.

Using the method of plasmapheresis repeatedly increases the potential of gene therapy, as this eliminates the obstacle called immune response of the organism.
As gene therapy becomes more widespread, it may be necessary, patients receiving more than one course of treatment.
The main problem is that when you go home after the first treatment, their body develops antibodies to the virus used to deliver the therapeutic gene. The use of plasma in a patient who previously received gene therapy may allow him to be treated again.

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