Alfredo A. Sadun, MD, PhD, and colleagues conducted an international post-mortem study of human eyes treated with adeno-associated virus (AAV2)-based gene therapy for Leber hereditary optic neuropathy (LHON). The results were reported at the American Academy of Ophthalmology annual meeting in San Francisco.
The study takeaway, according to first author Dr Sadun, was that the results provided the first molecular and cellular evidence of successful gene transfection of retinal ganglion cells in humans. This effect, interestingly, was seen in both patient eyes despite the injection being only unilateral. Dr Sadun is from the Doheny Eye Institute, UCLA David Geffen School of Medicine, Los Angeles.