WORLD / Health
Gene therapy used to cure mice blindness
(AP)
Updated: 2007-05-23 15:33
BAR HARBOR, Maine - Researchers at the University of Florida and The
Jackson Laboratory in Maine say they used gene therapy to restore sight
in mice with achromatopsia, a form of hereditary blindness that also
strikes humans.
In a paper published online in Tuesday's edition of Nature Medicine, the
Florida scientists working with Jackson research scientist Bo Chang
describe their use of a harmless virus to deliver corrective genes to
mice with a genetic impairment that deprives them of vision.
The discovery shows that it's possible to target and rescue cone cells,
the most important cells for visual sharpness and color vision in people.
The mice used in the research had achromatopsia, which affects one in
about 30,000 Americans by silencing cone photoreceptors in the retina.
The disease results in nearly complete color blindness and very poor
central vision.
"Cone vision defines whether someone is blind or not," said William
Hauswirth, professor of ophthalmic molecular genetics and a member of the
University of Florida Genetics Institute.
The ability to deliver a gene specifically to cone cells has implications
for all blinding diseases, not just inherited ones, Hauswirth said. "Even
in two very common types of blindness, age-related macular degeneration
and diabetic retinopathy, if you can target cones you might be able to
rescue that vision," he said.
Chang said the mouse with achromatopsia was found about three years ago
while screening many strains of mice for vision problems as part of The
Jackson Laboratory's program to develop new mouse models for human eye
conditions.
Chang and Jackson colleagues then identified the genetic mutation that
caused achromatopsia, while scientists elsewhere were zeroing in on the
analogous human mutation.
Within two months of the gene therapy injection, scientists measured the
electrical activity in the retinas. They found that 19 of the 21 treated
eyes responded positively to therapy; 17 of those 19 had electrical
readings from their retinas on par with those taken in normal mice.
Richard Weleber, professor of molecular and medical genetics at Oregon
Health and Science University, said the Florida-Jackson Lab research is
the first to his knowledge of a cone-targeted gene therapy that restores
function in an animal model when cones are the primary defect.
"This validates the concept that it is possible to deliver a gene therapy
targeting the cone system, and that is incredibly important for a number
of degenerative diseases," said Weleber, who was not involved in the
research.
The research was supported by grants from the National Institutes for
Health, the Juvenile Diabetes Research Foundation and other organizations.
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