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Methods Mol Biol. 2019;1937:281-294. doi: 10.1007/978-1-4939-9065-8_18.

Systemic Delivery of Adeno-Associated Viral Vectors in Mice and Dogs.

Wasala LP1, Hakim CH2,3, Yue Y2, Yang NN3, Duan D4,5,6,7,8.

Author information

1
Department of Veterinary Pathobiology, College of Veterinary Medicine, The University of Missouri, Columbia, MO, USA.
2
Department of Molecular Microbiology and Immunology, School of Medicine, The University of Missouri, Columbia, MO, USA.
3
National Center for Advancing Translational Sciences, NIH, Rockville, MD, USA.
4
Department of Veterinary Pathobiology, College of Veterinary Medicine, The University of Missouri, Columbia, MO, USA. duand@missouri.edu.
5
Department of Molecular Microbiology and Immunology, School of Medicine, The University of Missouri, Columbia, MO, USA. duand@missouri.edu.
6
Department of Neurology, School of Medicine, The University of Missouri, Columbia, MO, USA. duand@missouri.edu.
7
Department of Bioengineering, The University of Missouri, Columbia, MO, USA. duand@missouri.edu.
8
Department of Biomedical Sciences, College of Veterinary Medicine, The University of Missouri, Columbia, MO, USA. duand@missouri.edu.

Abstract

Many diseases affect multiple tissues and/or organ systems, or affect tissues that are broadly distributed. For these diseases, an effective gene therapy will require systemic delivery of the therapeutic vector to all affected locations. Adeno-associated virus (AAV) has been used as a gene therapy vector for decades in preclinical studies and human trials. These studies have shown outstanding safety and efficacy of the AAV vector for gene therapy. Recent studies have revealed yet another unique feature of the AAV vector. Specifically, AAV can lead to bodywide gene transfer following a single intravascular injection. Here we describe the protocols for effective systemic delivery of AAV in both neonatal and adult mice and dogs. We also share lessons we learned from systemic gene therapy in the murine and canine models of Duchenne muscular dystrophy.

KEYWORDS:

AAV; Adult dogs; Adult mice; DMD; Neonatal dogs; Neonatal mice; Systemic delivery

PMID:
30706404
DOI:
10.1007/978-1-4939-9065-8_18
[Indexed for MEDLINE]

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