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Production of Adeno-Associated Virus Vectors in Cell Stacks for Preclinical Studies in Large Animal Models
Published on: June 30, 2021
Large animal models for foamy virus vector gene therapy
Grant D Trobridge1, Peter A Horn, Brian C Beard
1Department of Pharmaceutical Sciences, Washington State University, 99164-6534, Pullman, WA, USA. grant.trobridge@wsu.edu
Viruses
|December 11, 2012
Summary
Foamy virus (FV) vectors show promise for hematopoietic stem cell (HSC) gene therapy in large animals. These vectors efficiently deliver genes, correct genetic disorders, and appear safer than other viral vectors for treating human blood diseases.
Area of Science:
- Gene Therapy
- Hematopoietic Stem Cells
- Retroviral Vectors
Background:
- Foamy virus (FV) vectors are being explored for hematopoietic stem cell (HSC) gene therapy.
- Their efficacy in large animal models suggests potential for human hematopoietic diseases.
Purpose of the Study:
- To review FV vector studies in large animal models for HSC gene therapy.
- To evaluate the safety and efficiency of FV vectors compared to other viral vectors.
Main Methods:
- Review of studies using FV vectors in large animal models.
- Assessment of gene transfer efficiency and safety profiles.
- Comparison with gammaretroviral and lentiviral vectors.
Main Results:
- FV vectors efficiently transduced multi-lineage, long-term repopulating cells.
- Short ex vivo transduction protocols were effective, minimizing negative impacts on engraftment.
- FV vectors corrected genetic defects in canine models (leukocyte adhesion deficiency, pyruvate kinase deficiency).
- FV vectors demonstrated a reduced frequency of risky integrants compared to gammaretroviral vectors.
Conclusions:
- FV vectors are highly effective for HSC gene therapy in large animal models.
- They offer advantages over gammaretroviral and lentiviral vectors in efficiency and safety.
- FV vectors hold significant potential for treating various human hematopoietic diseases.
