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Gene therapy helps arthritis pain, damage in mice
May 25, 2007
(Reuters) - A gene therapy treatment that helped make cells more
sensitive to the body's own painkillers not only helped ease arthritis
pain in mice but also reduced other symptoms, researchers said on
The researchers are now trying to get
permission to try the treatment in people.
"This therapy can simply be injected
anywhere in an injured joint, and the treatment will find the nerve
endings," said Dr. Stephanos Kyrkanides of the University of Rochester
Medical Center in New York, who led the study.
researchers used a well-tested method of gene therapy, employing a
virus called an adeno-associated virus to carry a new gene into the
body. In this case, the new gene was one called the human mu-opioid
receptor -- a kind of molecular doorway that the body's natural
painkillers use to get into cells.
The more of them a cell has, the more
sensitive it is to these painkilling chemicals.
The Kyrkanides team had mice genetically
engineered to develop osteoarthritis in the same way that people do.
They injected their gene-engineered virus into various joints.
"That strategy not only significantly
reduced the pain behavior in the mice with arthritis, but it also
helped to minimize the arthritis pathology itself," Kyrkanides said.
This suggests that the pain receptors play
a role not only in the pain but also in the joint damage caused by
arthritis, he said.
FOR HUMAN THERAPY
Osteoarthritis affects close to 10 percent
of men over the age of 60 and 18 percent of women over 60 worldwide,
according to the World Health Organization. Aspirin and other
analgesics can help, but they have often-deadly side effects.
Kyrkanides said he believes his gene
therapy system would work in people, as all mammals have the same
opiate receptor system.
"We used the actual human gene in our
therapy," he said. "I am certain that if the human gene had a
therapeutic effect in mice, it will certainly have it in humans as
Gene therapy has a mixed record. The idea
is to replace faulty genes, or to amplify the effects of genes that
people already have.
But many experiments have failed to show
lasting effects in people.
In addition, one 18-year-old gene therapy
volunteer was killed by an immune reaction to the virus that carried
the gene, and two babies given gene therapy for a rare immune condition
developed leukemia -- although they were successfully treated for the
Kyrkanides said his approach may be safer
because the virus is injected into the joint rather than infused into
an artery for delivery throughout the body.
"The great advantage of this is that it is
confined to an encapsulated area of the body," he said. "It does not
involve the whole body, thereby minimizing any general adverse effects
of the gene therapy itself."
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