News Review
Gene Therapy May Prevent
Retinoschisis in Boys
njecting a healthy version
I of a human gene may suc-
cessfully treat X-linked
juvenile retinoschisis (RS)—a
formerly untreatablc, inheri-
table eye disease that leads to
blindness in males—and
eventually other genetic eye
diseases that involve the eye's
ability to process light,
including retinitis pigmen-
tosa., according to a study
published in the August issue
of Molecular Therapy.
RS is most often identified
in boys between the ages of 5 Retinoschisis can cause total vision loss.
and 10 as a result of reported
reading problems. It is character-
ized by cystic spoke-wheel-like mac- had cyst formation.
ulopathy, peripheral schisis and a
negative electroretinogram (ERG).
In a normal eye, a protein called
retinoschisin., or RSI, is secreted
from retinal cells to hold the layers ution
of the retina together. Those with
RS do not have a functional RS1
protein, RS can cause total vision
loss later in life due associated com-
plications, such as choroidal sclero- nected in treated eyes.
sis, vitreous hemorrhage and retinal
detachment.
To combat RS., scientists from
the University of Florida Genetics
Institute, in Gainesville, Fla.,
injected a normal version of the
human RSI gene into the sub-reti-
nal space of the right eyes of 15-
day-old mice that lacked the normal ogy. "But we feel, based on success
glue-like gene. Their untreated left
eyes were used as controls. The
extent of RS in the mice was about
equal to the state of the disease
development in a 10-year-old boy.
On six-month follow-up, results
showed:
• The treated eyes appeared
10 REVIEW OF OPTOMETRY oEPTEMBEH 1 5, 2005
Jobson
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• RSI restored rod- and cone- (610) 4 9 2 - 1 0 1 1 • PMCMI-NAM1N@|OHSON,CDM
mediated ERG responses. CIRCULATION MANAGER
• Injection of the healthy human NORA PASTENKOS
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gene restored normal retinal distrib-
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When may a treatment become
clinically available? "It may take
two to five years before we try this J
lobson Publiihing. LLC
in human patients because of the
PRESIDENT
need for safety studies," says UMAS KHAKI I
William Hauswirth, Ph.D., one of PflESIDENT/iOBSON OPTICAL WORLDWIDE
the researchers and professor of MAKC FI.KRAKA
molecular genetics and microbiol- VICE PRESIDENT. HUMAN RESOURCES
so far, we will be able to provide VICE PRESIDENT, OPERATIONS DIRECTOR
IHDIIIi MiLHAH
formal evidence for safety that will
VICE PRESIDENT. CREATIVE DIRECTOR
allow us to get treatment into the RALPH S T F I I O
clinic." VICE PRESIDENT. CIRCULATION DIRECTOR
RMELDA BAREA
Min. SH. Molday, LL. Seeliger, MW, et al. Prolonged recov-
ery of retinal structure/tunction after gene Itierapy in an
Rsih-deficienI mouse model of X-linkefl juvenile retinosctii-
sis.Mol"nier2OO5Jul15:1-a,