Basic Concepts in Viral Vector-Mediated Gene Therapy

Authors

Matthew J. Benskey, Department of Translational Science and Molecular Medicine, Michigan State University, Grand Rapids, MI, USA.
Ivette M. Sandoval, Department of Translational Science and Molecular Medicine, Michigan State University, Grand Rapids, MI, USA.
Kathryn Miller, Department of Translational Science and Molecular Medicine, Michigan State University, Grand Rapids, MI, USA.
Rhyomi L. Sellnow, Department of Translational Science and Molecular Medicine, Michigan State University, Grand Rapids, MI, USA.
Aysegul Gezer, Department of Translational Science and Molecular Medicine, Michigan State University, Grand Rapids, MI, USA.
Nathan C. Kuhn, Department of Translational Science and Molecular Medicine, Michigan State University, Grand Rapids, MI, USA.
Roslyn Vashon, Department of Translational Science and Molecular Medicine, Michigan State University, Grand Rapids, MI, USA.
Fredric P. Manfredsson, Department of Translational Science and Molecular Medicine, Michigan State University, Grand Rapids, MI, USA. Fredric.manfredsson@hc.msu.edu.Follow

Document Type

Article

Abstract

Today any researcher with the desire can easily purchase a viral vector. However, despite the availability of viral vectors themselves, the requisite knowledge that is absolutely essential to conducting a gene therapy experiment remains somewhat obscure and esoteric. To utilize viral vectors to their full potential, a large number of decisions must be made, in some instances prior to even obtaining the vector itself. For example, critical decisions include selection of the proper virus, selection of the proper expression cassette, whether to produce or purchase a viral vector, proper viral handling and storage, the most appropriate delivery method, selecting the proper controls, how to ensure your virus is expressing properly, and many other complex decisions that are essential to performing a successful gene therapy experiment. The need to make so many important decisions can be overwhelming and potentially prohibitive, especially to the novice gene therapist. In order to aid in this challenging process, here we provide an overview of basic gene therapy modalities and a decision tree that can be used to make oneself aware of the options available to the beginning gene therapist. This information can be used as a road map to help navigate the complex and perhaps confusing process of designing a successful gene therapy experiment.

Keywords

Adeno-associated virus, Adenovirus, Gene therapy, Herpes-simplex virus, Lentivirus, Viral vector

Medical Subject Headings

Adenoviridae (genetics); Animals; Decision Making; Dependovirus (genetics); Gene Expression; Genetic Therapy (methods); Genetic Vectors (administration & dosage, physiology); Humans; Lentivirus (genetics); Simplexvirus

Publication Date

1-1-2019

Publication Title

Methods in molecular biology (Clifton, N.J.)

E-ISSN

1940-6029

Volume

1937

First Page

3

Last Page

26

PubMed ID

30706387

Digital Object Identifier (DOI)

10.1007/978-1-4939-9065-8_1

Recommended Citation

Benskey, Matthew J.; Sandoval, Ivette M.; Miller, Kathryn; Sellnow, Rhyomi L.; Gezer, Aysegul; Kuhn, Nathan C.; Vashon, Roslyn; and Manfredsson, Fredric P., "Basic Concepts in Viral Vector-Mediated Gene Therapy" (2019). Translational Neuroscience. 2016.
https://scholar.barrowneuro.org/neurobiology/2016