Optimized Subretinal Injection Technique for Gene Therapy Approaches

Abstract

Gene therapy for inherited eye diseases requires local viral vector delivery by intraocular injection. Since large animal models are lacking for most of these diseases, genetically modified mouse models are commonly used in preclinical proof-of-concept studies. However, because of the relatively small mouse eye, adverse effects of the subretinal delivery procedure itself may interfere with the therapeutic outcome. The method described here aims to provide the details relevant to perform a transscleral pars plana virus-mediated gene transfer to achieve an optimized therapeutic effect in the small mouse eye.

Keywords: Ablation; Bleb formation; Gene delivery; In vivo imaging techniques (SLO SD-OCT); Mouse eye; Photoreceptor cell; Recombinant adeno-associated virus; Retina; Subretinal injection; Transscleral pars plana injection.