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Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
Published on: August 10, 2018
Adeno-Associated Virus Delivery of Viral Serpins for Ocular Diseases: Design and Validation
Cristhian J Ildefonso1, Alfred S Lewin2
1Department of Ophthalmology, University of Florida College of Medicine, Gainesville, FL, USA. ildefons@ufl.edu.
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.
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