A friend sent me the following article that appeared on the
first page of the November 19th issue of the Times-Union
newspaper, from Rochester, N.Y.
"New Weapon Targets Brain-borne Diseases:
UR team finds that viruses can escort beneficial genes into
tissue, possibly reversing illnesses."
"Scientists at the University of Rochester and elsewhere are
exploring a new way to launch cell-size sneak attacks on parts of
the brain that cause degenerative diseases.
Doctors at a dozen medical centers internationally are
"infecting" cells with viruses wrapped around therapeutic genes,
which are then surgically implanted to act as long-lasting
medicine.
Within five years, gene delivery systems - already
successful in animal experiments - could be used on humans,
testing ways to reverse brain diseases like Alzheimer's and
Parkinson's, according to the National Institute of Mental
Health. Transported genes could also someday help eliminate brain
tumors and protect nervous systems battered by injury and stroke.
Virus-aided gene delivery promises to be safer, less toxic
and longer lasting than conventional drugs, researchers announced
today at an annual meeting of neuroscience scholars in Washington
D.C. Viruses for gene transfer is one of only 17 topics - out of
15,000 papers being presented - that merited a public symposium.
Co-chair od the event is Dr. Howard J. Federoff, 43, a UR
professor of neurology and medicine, microbiology and immunology
and chief of UR's division of molecular medicine and gene
therapy. He and other UR researchers are exploring how genes and
their carrier viruses - called "viral vectors" - interact with
host cells.
Viral vectors, said Federoff, are more efficient and
accurate at delivering genes to troubled nerve cells. Working
with a stripped down version of the same herpes virus that causes
cold sores, Federoff and his team can transfer large helper
proteins to nerve cells.
Their work has implications in restoring and preserving
nerve function in Parkinson's disease. About 1 in every 200
Americans are affected by this brain disorder, caused by damaged
nerve cell clusters in the brain.
If researchers can get delivered genes to (traits?) for up
to a year, said Federoff, their therapeutic effects may last a
lifetime. To date, researchers have hit the six-month mark.
Virus-based gene carriers include benign forms of viruses
linked to the common cold, cold sores and HIV, the virus that
causes AIDS. The smallest organisms known, viruses are protein-
coated packages of genetic material.
Viruses cannot eat or reproduce on their own, so they
reproduce by infecting plants and animals. Docking to the outside
of cells, viruses then penetrate cell walls and inject their
genetic material.
That makes them biology's cagiest infectious agents - ideal
candidates for carrying potentially "good" genes deep into the
cells of the brain, heart and other organs.
Hitchhiking on viruses, therapeutic genes are thought to
prop up diseased cells, washing through the body like a blood
transfusion.
Such genes can even be inserted into the body's finite
number of non-dividing cells, like those in the brain, nerves and
spinal cord.
The helper genes make the diseased cell more resistant or
help it function said Federoff. In general, gene therapy that
relies of virus-based "vectors" - transport systems - are
theoretically safer, less toxic, longer lasting and more reliable
than drug therapies, researchers said.
Especially promising in transporting helpful genes to nerve
cells is a lentiviral vector, based on HIV, a virus known for
being able to adhere to a wide variety of cells.
In gene transport therapy, viruses are disarmed by
technicians who snip away infectious traits. All that's left is
the virus's biological genius for docking on cell walls, then
wiggling its tail inside to inject genetic matter.
Therapeutic genes, hidden away in viruses, also boost the
efficiency of some conventional drug therapies, researchers
said."
Linda Herman
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