Gene therapy: Hope for the blind?

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Gene therapy is offering the potential to actually cure certain diseases and an experimental breakthrough could one day help blind patients see. (KFSN)

In medicine, doctors often talk about treatments, but rarely about cures. Now, gene therapy is offering the potential to actually cure certain diseases and an experimental breakthrough could one day help blind patients see.

Six-year-old Andy Moorhead is learning how to read. But instead of using his eyes, he's using his fingers. Andy told ABC30, "Well, I read the letters with my fingers."

Andy is blind. Andy's Mother, Heather Ingram-Moorhead explained, "He was around nine months, and we started to notice his eyes were twitching."

Andy has leber congenital amaurosis, or LCA. It's the most common type of childhood blindness and is caused by genetic mutations.

"It is just very hard. It's taken us a while to really understand the condition and do everything to help Andy," Heather told ABC30.

Andy's whole family is hands-on. Even his sister Valerie gives him guidance. But despite their efforts, his mom says gene therapy is their only hope.

University of Florida scientist Shannon E. Boye, PhD, is using a $900,000 grant to perfect a gene therapy that could restore vision.

"It's not an attempt just to slow the progression of the disease. It's actually an attempt to halt the progression and make the patient better by delivering them the gene they don't have," Boye told ABC30.

Boye says the therapy has worked in animals. "We're able to show, via what's called an electra retinal gram, that the retinal function has been restored to the mice," she explained.

Gene therapy is still an investigational treatment with risks and only available for those in a clinical trial. Right now there are hundreds of studies underway to treat conditions like LCA, cancer and HIV. It's hope that one day Andy could put down his cane and see his family for the first time.

Dr. Boye says gene therapy is typically a one-time treatment. According to the National Institutes of Health, gene therapy in the eye has a good safety record so far and is still under study to make sure it is safe and effective. You can search for clinical trials involving gene therapy online at clinicaltrials.gov.

For more information, contact:

Rossana Passaniti, CMP
Media Relations Coordinator
UF Health
352-273-8569
passer@shands.fl.edu



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