Optimized Subretinal Injection Technique for Gene Therapy Approaches.

Optimized Subretinal Injection Technique for Gene Therapy Approaches.
Methods Mol Biol. 2019;1834:405-412
Authors: Mühlfriedel R, Michalakis S, Garrido MG, Sothilingam V, Schön C, Biel M, Seeliger MW
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.
PMID: 30324458 [PubMed - in process]

Source: Mol Biol Cell - October 17, 2018 Category: Molecular Biology Authors: Mühlfriedel R, Michalakis S, Garrido MG, Sothilingam V, Schön C, Biel M, Seeliger MW Tags: Methods Mol Biol Source Type: research