Published: Wednesday, July 24, 2013 at 4:59 p.m. Last Modified: Wednesday, July 24, 2013 at 4:59 p.m.

The University of Floridas ophthalmology department, together with gene therapy company AGTC in Alachua, received an $8.4 million grant from the National Eye Institute at the National Institutes of Health to study gene therapy for achromatopsia, a genetic condition that severely impairs peoples vision.

The NEI gives out few grants each year, and this one lasts five years.

Its a very significant grant. Its a great validation of the team that we put together, said Sue Washer, president and CEO of AGTC.

According to Dr. William Hauswirth, the UF professor who will be leading the UF portion of the trial, Barring any real unforeseen and unfortunate challenges, well be trying it on patients in three years.

Hauswirth has been working on gene therapy for blindness for the past 30 years, using recombinant viruses to generate copies of faulty or missing genes affecting vision.

Achromatopsia, which affects the cones on the retina called fovea, makes it very difficult for people to read, navigate and recognize others. Patients are also very sensitive to bright room light, and most are legally blind, Hauswirth explained.

Its a relatively common disease, even though its still considered an orphan disease, but there are lots of patients out there, Hauswirth said, adding that the condition affects about 22,000 people in the United States and Europe.

They would all love to do (the trial), but well have to start carefully with a few patients in each site.

Washer said the therapy being studied in this trial could potentially help about half those patients, those who carry the faulty gene.

Read more from the original source:
Researchers see gene-therapy grant improving vision

Comments are closed.