The Bascom Palmer Eye Institute of the University of Miami Miller School of Medicine has released long-term results of a gene therapy clinical trial in the U.S. for Leber Hereditary Optic Neuropathy (LHON), which showed low and medium doses improved visual acuity in most patients when treated within one year of onset of visual loss.
The study was reportedly initiated in 2014 and involved 14 patients with the mitochondrial disease LHON. Through the trial, patients with vision loss from LHON reportedly received a single-unilateral dose injection of a normal version of their mutated mitochondrial gene into the eye. According to researchers, acuity begin to improve within one month of treatment and continued to do so as long as 18 months later.
Additionally, OCT measurements of the retinal nerve fibers reportedly showed treatment halted the characteristic degeneration of the optic nerve that continued to deteriorate in untreated eyes at 12 months of follow-up.
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Source: Bascom Palmer Eye Institute