RNA Biology in Retinal Development and Disease

For decades, RNA has served in a supporting role between the genetic carrier (DNA) and the functional molecules (proteins). It is finally time for RNA to take center stage in all aspects of biology. The retina provides a unique opportunity to dissect the molecular underpinnings of neuronal diversity and disease. Transcriptome profiles of the retina and its resident cell types have unraveled unique features of the RNA landscape. The discovery of distinct RNA molecules and the recognition that RNA processing is a major cause of retinal neurodegeneration have prompted the design of biomarkers and novel therapeutic paradigms. We review here RNA biology as it pertains to the retina, emphasizing new avenues for investigations in development and disease. After decades of playing second fiddle, RNA has now acquired its coveted role in all aspects of biological research. Transcriptome profiling has identified key features unique to the retina, including non-coding RNAs that are likely linked to both development and disease pathology. Alternative splicing is a hallmark of transcriptomes of developing mammalian retina, and especially of photoreceptors. At least 50% of the retina-expressed genes exhibit an altered structure compared to the reference. Diverse and functionally characterized neurons in the retina provide a unique model to dissect the function of distinct RNA molecules in biological processes and evaluate novel therapeutic paradigms.