More Success With Gene Therapy for Blindness

WEDNESDAY, Feb. 8 (HealthDay News) — As a child, Tami Morehouse
had vision problems. She struggled to read the blackboard at school, and
homework took hours.

Yet, she made it through high school and college, and became a social
worker. Although she was never able to drive, she learned to ride a
bike.

But in her 30s, with three young children, her vision took a turn for
the worse. “I’d be reading a book and the words faded away,” she said.

Morehouse was going blind, the result of Leber congenital amaurosis
(LCA), a rare inherited eye disease that causes a progressive loss of
vision. “As my kids needed me more and more, I was able to do less and
less,” Morehouse said.

That changed in 2009, when she was one of 12 people to undergo an
experimental treatment using gene therapy in one eye. Now, scientists
report even more progress, having successfully treated the second eye of
three patients, including Morehouse. The new results are published Feb. 8
in Science Translational Medicine.

LCA is caused by a faulty gene, RPE65, that fails to produce an enzyme
needed by the retina, the tissue in the back of the eye that converts
light images into nerve signals that get sent to the brain.

Lack of the enzyme causes toxic byproducts to build up in the retinal
cells, gradually killing them.

“It’s inevitable and progressive, and people watch as they are losing
more and more of their vision,” said Dr. Jean Bennett, an ophthalmology
professor at University of Pennsylvania Perelman School of Medicine and
co-leader of the research team that pioneered the treatment. “By the time
they’re teenagers or young adults, they are severely impaired.”

The treatment involved injecting a virus genetically engineered to
carry a normal version of gene RPE65 into the retinal cells.

About two weeks later, with the eyes now producing the enzyme, the
patients — adults and children — saw a marked improvement in their
vision.

“They all gained vision in a very meaningful way,” said Bennett, also a
scientist at Children’s Hospital of Philadelphia. “Children can read
books, ride their bikes to their friends’ houses — things which they
never could do before.”

Initially, researchers only injected one eye because of safety
concerns, Bennett explained. The fear was that the first injection would
prime the immune system to recognize the virus and attack it when it was
injected into the second eye. That would cause inflammation in the eye,
potentially leading to more vision loss.

But animal studies showed that didn’t happen, and so they decided it
was safe to try the second eye in adults.

“It’s amazing,” Morehouse said. “I just feel so different. I used to
wake up in the morning, so afraid and so anxious, that I would look over
at the alarm clock and see nothing.”

Prior to the treatments, she could see light and dark, but most of the
world was hazy and gray. By night time, when her eyes were tired, she
could see very little.

Today, her vision is still significantly impaired. She needs help
finding her way to a table in a restaurant, for example, and reading isn’t
really possible. Yet, she can tell when someone is approaching, and she
can make out a smile.

“Seeing my daughter walk across the basketball court. Seeing my son
step up to the plate when he’s playing ball — it’s phenomenal,” Morehouse
said.

Researchers verified that patients could see more by performing
functional MRI scans before and after the second eye treatment. The brain
imaging showed much more response to visual stimuli after their second eye
was done.

At 47, Morehouse was the oldest patient. The prior study taught
researchers that children improved the most, probably because their
retinal cells had suffered less damage.

Now that researchers have established the procedure is safe in adults,
they’ve started using gene therapy on the second eyes of children with the
condition, Bennett said.

Though more research needs to be done and there are “technical” issues
to be overcome, “we want to be able to use this approach in developing
similar treatments for other more common blinding diseases,” she said.

More information

The Foundation Fighting Blindness has more on LCA.

Views: 0

You can skip to the end and leave a response. Pinging is currently not allowed.

Leave a Reply

Powered by WordPress | Designed by: Premium WordPress Themes | Thanks to Themes Gallery, Bromoney and Wordpress Themes