Originally published October 27 2005
Pittsburgh researchers successfully deliver therapeutic gene
by Mike Adams, the Health Ranger, NaturalNews Editor
University of Pittsburgh researchers successfully used gene therapy to treat heart failure associated with muscular dystrophy.
"Previous attempts at systemic gene therapy for muscle have not been very effective because blood vessel capillaries act much like a mosquito net, blocking the gene drugs from reaching the muscle cells.
Fortunately, we found a virus that is just small and sneaky enough to get through this net and deliver the therapeutic gene to both skeletal and cardiac muscle cells," said lead author, Xiao Xiao, Ph.D., associate professor of orthopaedic surgery at the University of Pittsburgh School of Medicine.
The virus used by Dr. Xiao and his colleagues for delivering the corrective gene is known as adeno-associated virus, or AAV, a class of relatively small viruses that do not cause any known disease.
In earlier studies, Dr. Xiao's team found that direct intramuscular injection of AAV was effective in transferring a gene into muscle cells in a fairly wide area around the injection site.
In human LGMD, defects in a muscle cell membrane protein known as delta-sarcoglycan lead to severe damage and weakness to muscles, particularly around the hips and shoulders--hence the name "limb girdle"-- as well as in the heart.
Like humans, hamsters with this particular delta-sarcoglycan gene defect have severe muscle wasting and weakness and significantly shortened lifespans due to cardiac and respiratory failure.
After injecting a very high dose of AAV-8 carrying a normal copy of the delta-sarcoglycan gene intravenously into 10-day-old and adult LGMD hamsters, Dr. Xiao and his colleagues found that it had been systemically incorporated into skeletal, diaphragm and cardiac muscle cells in both groups.
Because 30 percent to 40 percent of the population has antibodies to human AAVs, there is always the possibility that the effectiveness of this form of gene therapy may be blunted by a host immune response.
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