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<DIV><FONT face=Arial size=2>I saw this on Fox news and thought it was very
interesting. I wonder if this experiemtn could work with people with retinopathy
of prematurity or with only genetic causes of blindness.</FONT></DIV>
<DIV><FONT face=Arial size=2>Sherri</FONT></DIV>
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<H1>Study Suggests Gene Therapy May Enable Blind to See</H1>
<P><STRONG>Wednesday, August 12, 2009</STRONG></P>
<H4>By Marrecca Fiore</H4>
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<P>Scientists believe the brain of a blind woman rewired itself and enabled her
to see after partial vision was restored to sections of her retina through gene
therapy.</P>
<P>The finding surprised scientists at the University of Florida College of
Medicine and the University of Pennsylvania who two years ago began testing the
safety of gene therapy in three patients born with Leber congenital amaurosis
type 2, a rare blindness condition. The findings are contained in a study to be
published Thursday in the New England Journal of Medicine.</P>
<P>“This was phase 1 of a National Eye Institute study and we wanted to measure
whether gene therapy was safe for the eye and safe for the body,” said Dr. Artur
Cideciyan, a research associate professor at the University of Pennsylvania. “We
were hopeful that something positive would end up occurring as well.”</P>
<P>Scientists found that within weeks of adding healthy genes to the retinas of
study participants (a woman and two men between the ages of 21 and 24), the
three began to experience significant improvements, including improved
sensitivity to light.</P>
<P>“We found that the improvements occurred only in the treated eyes and in the
regions that were injected with the new genes,” Cideciyan said. “We also found
that the improvements and the safety were maintained for 12 months after the
initial injection.”</P>
<P>The tiny portions of the patients’ retinas that received gene therapy
experienced restored function up to 1,000-fold during the day and 63,000-fold at
night.</P>
<P>But it was the woman in particular who surprised researchers when she
announced that a year after receiving treatment, her vision continued to make
small improvements. She said she could read the digital clock in her parents’
car – something she had never done before.</P>
<P>“That prompted us to measure where her gaze was fixed while looking at a
variety of dim targets,” said Dr. William W. Hauswirth, a professor in the
ophthalmology department at the UF College of Medicine, who led the study. “This
showed she now has two preferred centers of vision rather than one, depending on
the brightness of the object.”</P>
<P>Cideciyan said healthy adults have a single preferred center of vision that
they use to see objects. Based on their findings, researchers hypothesize that
the female patient’s brain rewired itself to use two preferred centers, which it
shifts back and forth between depending on the situation.</P>
<P>“I think that it is a very intriguing hypothesis … that she learned to use an
area of her retina that was improved through gene therapy,” said Dr. Brian
Brooks, an investigator with the National Institute of Health’s National Eye
Institute.</P>
<P>“We don’t yet understand the precise mechanism that occurred to make this
happen,” said Brooks, who did not participate in the NEI-supported study. “But
the hope is that we will be able to use the finding to cold train patients to
use new areas of their retinas that they’re not accustomed to using.”</P>
<P>The finding could also prove beneficial to others with degenerative retinal
conditions similar to Leber congenital amaurosis, which is a leading cause of
blindness in infants and children, Cideciyan and Brooks said.</P>
<P>Cideciyan said the safety trial will continue for the next three years and
the participants will be followed for 15 years, as mandated by the U.S. Food and
Drug Administration. The study also will be expanded to include other age groups
and participants.</P><BR style="CLEAR: both">
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