Macula Vision Research Foundation Spring/Summer 2017 7 Dwight Stambolian, mD, PhD Associate Professor Department of ophthalmology and human genetics University of Pennsylvania SingLe CeLL SeqUenCing of retinAL CeLLS Advances in our understanding of AMD have been limited due to inadequate animal models and lack of postmortem human eye tissue. Some of the next advances in our understanding of AMD will require building upon the information provided by recent genetic studies on DNA published by us and others by characterizing RNA in normal and AMD human eyes. Our previous work on RNA has found significant differences between the macula and non-macula regions of the retina suggesting that these differences could provide insight on why AMD is limited to the macula region. To understand how these differences affect cell behavior we propose to focus on characterizing the RNA genome of retinal cells in AMD and normal eyes. Our results could lead to new cell-specific therapeutic targets and stimulate research linking genes to cellular pathology. Samuel g. Jacobson, mD, PhD Director, Center for hereditary retinal Degenerations Director, retinal function Department Scheie eye institute, University of Pennsylvania CLiniCAL triALS of Severe Cone PhotoreCePtor DiSeASeS: A noveL moBiLity PerformAnCe oUtCome We have entered an era in which treatment of previously incurable and untreatable retinal degenerations has become a realistic goal. Now we need quantitative and accurate techniques to assess ‘real-life’ efficacy in clinical trials. Specifically, there has been progress in understanding and planning treatments for cone-specific retinal diseases that begin in childhood and involve the macula profoundly. There is thus a need to devise and implement a ‘cone mobility performance task’ and this is the goal of this proposal. We have already tested the hypothesis at the basis of the devising and using of a ‘cone mobility performance task’ and we proved the feasibility of this idea in a subset of patients and in normal subjects. Now, we will further develop the concept, build the course and determine inter-visit variability in order to use it as an assay in clinical trials of treatment.