Adeno-Associated Virus Delivery of Viral Serpins for Ocular Diseases: Design and Validation.

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TitleAdeno-Associated Virus Delivery of Viral Serpins for Ocular Diseases: Design and Validation.
Publication TypeJournal Article
Year of Publication2018
AuthorsIldefonso, CJ, Lewin, AS
JournalMethods Mol Biol
Volume1826
Pagination237-254
Date Published2018
ISSN1940-6029
Abstract

Adeno-associated virus (AAV) has become the preferred viral gene transfer platform for ocular gene therapy due to its known safety profile in human clinical trials. This viral vector has a 4.7 kbp (kilo base pair) carrying capacity (single-stranded DNA) and only retains the inverted terminal repeats (ITRs) from the original virus. Here we describe the design and testing of AAV vectors capable of delivering an anti-inflammatory serine protease inhibitor (serpin) derived from the myxoma virus. Myxoma is a rabbit species specific virus infection, a Leporipoxvirus. Myxomaviral proteins have been developed as therapeutic stand-alone immune-modulating proteins for inflammation-based disorders and the myxoma virus itself is under development as a viral oncolytic platform for cancer treatment. We fused the Serp2 gene with the GFP reporter gene through a self-cleaving peptide.

DOI10.1007/978-1-4939-8645-3_16
Alternate JournalMethods Mol. Biol.
PubMed ID30194605