Our lab is studying the molecular and genetic basis of Usher syndrome (USH; deaf-blindness) and oculocutaneous albinism (OCA), utilizing human, mouse, and zebrafish genetics. For these studies, large families segregating inherited USH and OCA are being collected. Mutant mouse and zebrafish models have been developed, and we are now evaluating them to understand the function of new genes. Functional analysis of the newly identified genes associated with deaf-blindness and OCA promises new insights into the molecular mechanisms of vision and auditory development. Identification of causative gene variants for vision and hearing loss, and concomitant discovery of molecular pathways that are disrupted during the said disorders have provided potential therapeutic targets. Future treatment of vision and deafness will inevitably include gene therapy and antisense delivery. We are using viral-based gene delivery protocols to rescue the hearing and vision loss in Usher syndrome mouse models. However, targeting and expression in a cell-type-specific manner is challenging and a limitation. Non-viral-based gene delivery vectors like nanoparticles are the most promising types of delivery systems that can be used for selective targeting. Jointly with my colleague Dr. Riazuddin at University of Maryland, we are also actively pursuing strategies based on various RNA nanoparticles for specific delivery of small molecules like siRNA and genes to targeted cells in the retinal and cochlear sensory epithelium.