Six-year-old Andy Moorhead is learning how to read, but instead of using his eyes, he's using his fingers.
"Well, I read the letters with my fingers," said Andy, who is blind.
"He was around nine months, and we started to notice his eyes were twitching," explained Andy's mother, Heather Ingram-Moorhead.
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," Ingram-Moorhead said.
Andy's whole family is hands-on. Even his sister, Valerie, gives him guidance, but despite their efforts, his mom said gene therapy is their only hope.
Shannon E. Boye, a scientist at the University of Florida, 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 said.
Boye said 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.
Boye said 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 online for clinical trials involving gene therapy.
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