Adeno-Associated Virus Delivery of Viral Serpins for Ocular Diseases: Design and Validation.
Submitted by Cristhian J Ildefonso on
Title | Adeno-Associated Virus Delivery of Viral Serpins for Ocular Diseases: Design and Validation. |
Publication Type | Journal Article |
Year of Publication | 2018 |
Authors | Ildefonso, CJ, Lewin, AS |
Journal | Methods Mol Biol |
Volume | 1826 |
Pagination | 237-254 |
Date Published | 2018 |
ISSN | 1940-6029 |
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. |
DOI | 10.1007/978-1-4939-8645-3_16 |
Alternate Journal | Methods Mol. Biol. |
PubMed ID | 30194605 |