title: Retinal gene delivery by adeno-associated virus (AAV) vectors: Strategies and applications.
abstract: Adeno-associated virus (AAV) vectors are the most widely used vehicle systems for neuronal gene transfer. This popularity is based on the non-pathogenic nature of AAVs and their versatility making them a multifunctional vector system for basic research and clinical applications. AAVs are successfully applied in clinical and pre-clinical gene therapy studies for inherited retinal disorders. Their excellent transduction profile and efficiency also boosted the use of AAV vectors in basic research. The AAV vector system can be easily modified and adjusted at multiple levels to allow for optimized and specific gene expression in target cells. Here, we will provide an overview on the AAV vector system and its applications focusing on gene transfer into retinal cells. Furthermore, we will outline and discuss strategies for the optimization of AAV gene transfer by modifications to the AAV vector expression cassette, the AAV capsid or the routes of vector administration.
authors.affiliation: Center for Integrated Protein Science Munich CiPSM, Department of Pharmacy - Center for Drug Research, Ludwig-Maximilians-Universität München, Munich, Germany. Electronic address: christian.schoen@cup.uni-muenchen.de.
authors.forename: Christian
authors.initials: C
authors.lastname: Schön
authors.affiliation: Center for Integrated Protein Science Munich CiPSM, Department of Pharmacy - Center for Drug Research, Ludwig-Maximilians-Universität München, Munich, Germany. Electronic address: mbiel@cup.uni-muenchen.de.
authors.forename: Martin
authors.initials: M
authors.lastname: Biel
authors.affiliation: Center for Integrated Protein Science Munich CiPSM, Department of Pharmacy - Center for Drug Research, Ludwig-Maximilians-Universität München, Munich, Germany. Electronic address: stylianos.michalakis@cup.uni-muenchen.de.
authors.forename: Stylianos
authors.initials: S
authors.lastname: Michalakis
mesh_terms: D000818:Animals; D000229:Dependovirus / Q000235:genetics*; D005786:Gene Expression Regulation; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000008:administration & dosage; D006801:Humans; D012160:Retina / Q000166:cytology / Q000378:metabolism; D012164:Retinal Diseases / Q000235:genetics / Q000628:therapy*
keywords: AAV capsid; AAV genome; Adeno-associated virus; Gene therapy; Intravitreal injection; Retina; Subretinal injection