funding, is renowned for having
developed some of the most effective gene-delivery systems known as
“You can think of a vector like a
taxi cab, and the therapeutic DNA
as the passenger inside,” Dr. Boye
explains. “We, as gene therapists,
give a cabbie directions on where
that cab should go and where to
drop the passenger off.”
While working in Dr. Hauswirth’s
lab, she met and eventually married
another member of his team—Dr.
Sanford Boye. They have two children—Sydney, 6, and Benjamin, 4.
And it’s their kids Dr. Boye thinks of
when parents plead with her to find
cures for retinal diseases.
Her Vision-Saving Work
Here are a few of the treatments Dr.
Boye is helping develop:
A gene therapy for Leber congen-
ital amaurosis caused by a defect
in the GUCY2D gene. This work is
not only supported by FFB; it was
recently awarded a $900,000 grant
by Genzyme, a pharmaceutical
company showing great interest
in moving the treatment toward
A gentler gene-delivery system.
Gene therapies require injections
delivered beneath the retina. But
because that retina is severely dam-
aged by disease, “it’s less likely to
withstand surgical manipulation,”
Dr. Boye says. The new approach
would deliver genes through the
vitreous, or front of the eye, qual-
ifying as an outpatient procedure.
“It would be a paradigm shift,”
A dual-vector delivery—meaning
using two taxis, not just one—so as
to replace genes that are currently
too big for conventional vectors. “In
pre-clinical studies, we’ve had a lot
of success delivering a healthy ver-
sion of Myosin7A, a large gene that
can cause Usher syndrome, which
results in both vision and hearing
loss,” Dr. Boye explains. “So, as you
can imagine, the potential is pro-
The last two projects are also
funded by FFB, which invests only
in those researchers whose work
shows great promise.
Dr. Boye and the colleagues
she’s aligned with meet that high
Hence the smaller (8-by-ll½-inch) format, the
colorful layout, the sprinkling of retinal and
FFB-related factoids and the shorter stories. But no
worries—we’re not short-changing you in terms
of information. As with past issues, this one offers
a variety of useful articles, some linked to online
resources. What we’re doing is turning In Focus into
a handy tool—one that will serve our collective
awareness- and fundraising efforts.
So, what’s in this issue? A page-one story on
multimedia efforts to share what vision loss looks like.
A down-to-earth profile of a cutting-edge researcher
(pg. 2). A map pinpointing FFB-funded researchers
and facilities worldwide (p. 4). Stories on our My
Retina Tracker™ registry (pg. 6), an extraordinary teen
with RP (pg. 7) and an enthusiastic fundraiser (pg. 7).
On page 4, you’ll also find a URL for an online survey
I encourage you to fill out. Your answers will help us
make In Focus an even better newsletter.
A new year, a new-and-improved publication and
an unflagging commitment, as always, to eradicate
retinal diseases. I can’t think of a better
way to kick off 2015!
It’s her kids Dr. Boye
thinks of when parents plead
with her to find cures for