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In-Vivo Gene Therapy with Foamy Virus Vectors

1
Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA
2
Departments of Medicine, University of Washington School of Medicine, Seattle, WA 98195, USA
3
Departments of Pathology, University of Washington School of Medicine, Seattle, WA 98195, USA
*
Author to whom correspondence should be addressed.
Viruses 2019, 11(12), 1091; https://doi.org/10.3390/v11121091
Received: 29 September 2019 / Revised: 20 November 2019 / Accepted: 20 November 2019 / Published: 23 November 2019
(This article belongs to the Special Issue Spumaretroviruses)
Foamy viruses (FVs) are nonpathogenic retroviruses that infect various animals including bovines, felines, nonhuman primates (NHPs), and can be transmitted to humans through zoonotic infection. Due to their non-pathogenic nature, broad tissue tropism and relatively safe integration profile, FVs have been engineered as novel vectors (foamy virus vector, FVV) for stable gene transfer into different cells and tissues. FVVs have emerged as an alternative platform to contemporary viral vectors (e.g., adeno associated and lentiviral vectors) for experimental and therapeutic gene therapy of a variety of monogenetic diseases. Some of the important features of FVVs include the ability to efficiently transduce hematopoietic stem and progenitor cells (HSPCs) from humans, NHPs, canines and rodents. We have successfully used FVV for proof of concept studies to demonstrate safety and efficacy following in-vivo delivery in large animal models. In this review, we will comprehensively discuss FVV based in-vivo gene therapy approaches established in the X-linked severe combined immunodeficiency (SCID-X1) canine model. View Full-Text
Keywords: gene therapy; in-vivo gene therapy; hematopoietic stem and progenitor cells; foamy virus vector; pre-clinical canine model; SCID-X1 gene therapy; in-vivo gene therapy; hematopoietic stem and progenitor cells; foamy virus vector; pre-clinical canine model; SCID-X1
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Rajawat, Y.S.; Humbert, O.; Kiem, H.-P. In-Vivo Gene Therapy with Foamy Virus Vectors. Viruses 2019, 11, 1091.

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