MicroRNA-Regulated Gene Networks in the Retina

The eye is one the main tissue targets of genetic diseases. Some estimates suggest that eye involvement is present in about 20% of all genetic diseases. Among the genetic conditions that affect primarily the eye, retinitis pigmentosa (RP) is one of the most frequent. RP has a frequency of about 1 in 3,500 individuals and leads to severe visual deficits and ultimately to blindness. In the past few years, significant progress was made on the recognition of the genetic causes of RP. Moreover, gene therapy-based approaches yielded initial encouraging results towards a possible therapeutic treatment of this group of disorders. Nevertheless, we are still far both from the complete elucidation of all the possible genetic causes of RP and from an effective cure in the majority of patients. Recently, some small fragments of the human genome, termed microRNAs, have been identified and found to have a fundamental role in many biological processes, both in physiological and in pathological conditions. The goal of this project is the study of the possible role of microRNAs both as causative agents and as therapeutic agents in retinitis pigmentosa.