Retinal Structure and Function in Achromatopsia: Implications for Gene Therapy

Conclusions: The lack of a clear association of disruption of retinal structure or function in ACHM with age suggests that the window of opportunity for intervention by gene therapy is wider in some individuals than previously indicated. Therefore, the potential benefit for a given subject is likely to be better predicted by specific measurement of photoreceptor structure rather than simply by age. The ability to directly assess cone photoreceptor preservation with SD-OCT and/or adaptive optics imaging is likely to prove invaluable in selecting subjects for future trials and measuring the trials' impact.

Source: Ophthalmology - October 21, 2013 Category: Opthalmology Authors: Venki Sundaram, Caroline Wilde, Jonathan Aboshiha, Jill Cowing, Colin Han, Christopher S. Langlo, Ravinder Chana, Alice E. Davidson, Panagiotis I. Sergouniotis, James W. Bainbridge, Robin R. Ali, Alfredo Dubra, Gary Rubin, Andrew R. Webster, Anthony T. Mo Tags: Original Articles Source Type: research