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.

• Authors: Regine Mühlfriedel, Stylianos Michalakis, Marina Garcia Garrido, Vithiyanjali Sothilingam, Christian Schön, Martin Biel, Mathias W Seeliger
• Journal: Methods in molecular biology (Clifton, N.J.) (Methods Mol Biol) Vol. 1834 Pg. 405-412 ( 2019) ISSN: 1940-6029 [Electronic] United States
• PMID: 30324458 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't)

Topics

• Animals
• Dependovirus (genetics)
• Gene Transfer Techniques
• Genetic Therapy (methods)
• Genetic Vectors (genetics)
• Humans
• Injections, Intraocular (methods)
• Mice
• Photoreceptor Cells (metabolism)
• Retina (cytology, metabolism)