Adeno‐associated virus‐mediated gene transfer
- 21 August 2008
- journal article
- prospect
- Published by Wiley in Journal of Cellular Biochemistry
- Vol. 105 (1), 17-24
- https://doi.org/10.1002/jcb.21819
Abstract
Although the remarkable versatility and efficacy of recombinant adeno‐associated virus 2 (AAV2) vectors in transducing a wide variety of cells and tissues in vitro, and in numerous pre‐clinical animal models of human diseases in vivo, have been well established, the published literature is replete with controversies with regard to the efficacy of AAV2 vectors in hematopoietic stem cell (HSC) transduction. A number of factors have contributed to these controversies, the molecular bases of which have begun to come to light in recent years. With the availability of several novel serotypes (AAV1 through AAV12), rational design of AAV capsid mutants, and strategies (self‐complementary vector genomes, hematopoietic cell‐specific promoters), it is indeed becoming feasible to achieve efficient transduction of HSC by AAV vectors. Using a murine serial bone marrow transplantation model in vivo, we have recently documented stable integration of the proviral AAV genome into mouse chromosomes, which does not lead to any overt hematological abnormalities. Thus, a better understanding of the AAV–HSC interactions, and the availability of a vast repertoire of novel serotype and capsid mutant vectors, are likely to have significant implications in the use of AAV vectors in high‐efficiency transduction of HSCs as well as in gene therapy applications involving the hematopoietic system. J. Cell. Biochem. 105: 17–24, 2008.Keywords
This publication has 73 references indexed in Scilit:
- Applying macromolecular crowding to enhance extracellular matrix deposition and its remodeling in vitro for tissue engineering and cell-based therapiesAdvanced Drug Delivery Reviews, 2011
- Collagen matrix physical properties modulate endothelial colony forming cell-derived vessels in vivoMicrovascular Research, 2010
- Evaluation of tissue components in the peripheral nervous system using Sirius red staining and immunohistochemistry: A comparative study (human, pig, rat)Journal of Neuroscience Methods, 2010
- Mechanical properties of completely autologous human tissue engineered blood vessels compared to human saphenous vein and mammary arteryBiomaterials, 2008
- Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower dosesProceedings of the National Academy of Sciences, 2008
- The Role of DNA-PKcs and Artemis in Opening Viral DNA Hairpin Termini in Various Tissues in MiceJournal of Virology, 2007
- Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapyJCI Insight, 2007
- Self-Complementary Recombinant Adeno-Associated Viral Vectors: Packaging Capacity And The Role of Rep Proteins in Vector PurityHuman Gene Therapy, 2007
- Vascular smooth muscle cell response on thin films of collagenMatrix Biology, 2005
- Adeno-associated virus vectors: potential applications for cancer gene therapyCancer Gene Therapy, 2005