Journal
EXPERT OPINION ON BIOLOGICAL THERAPY
Volume 9, Issue 11, Pages 1427-1436Publisher
TAYLOR & FRANCIS LTD
DOI: 10.1517/14712590903246388
Keywords
foamy virus; gene therapy; hematopoietic stem cell; retroviral vector; spumaretrovirus
Funding
- NIAID NIH HHS [R21 AI063959-02, R21 AI063959] Funding Source: Medline
- NIDDK NIH HHS [R21 DK077806-02, R21 DK077806] Funding Source: Medline
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Foamy virus (FV) vectors are efficient gene delivery vehicles that have shown great promise for gene therapy in preclinical animal models. FVs or spumaretroviruses are not endemic in humans, but are prevalent in nonhuman primates and in other mammals. They have evolved means for efficient horizontal transmission in their host species without pathology. FV vectors have several unique proper-ties that make them well suited for therapeutic gene transfer including a desirable safety profile, a broad tropism, a large transgene capacity, and the ability to persist in quiescent cells. They mediate efficient and stable gene transfer to hematopoietic stem cells (HSCs) in mouse models, and in the canine large animal model. Analysis of FV vector integration sites in vitro and in hematopoietic repopulating cells shows they have a unique integration profile, and suggests they may be safer than gammaretroviruses or lentiviral vectors. Here, properties of FVs relevant to the safety and efficacy of FV vectors are discussed. The development of FV vector systems is described, and studies evaluating their potential in vitro, and in small and large animal models, is reviewed.
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