Conclusions
AAV vectors have now proven to be very efficient for stable gene delivery into a number of in vivo targets. At this time, preparation of high quality and high titer rAAV, although dramatically improved over the past couple of years, remain a significant bottleneck. Yet, specifications for clinical grade material are starting to be defined and phase I clinical trials involving rAAV as vector are now underway. In animal experiments, high gene transfer efficiency is seen in myotubes and neurones, which are post-mitotic cells, and possibly in a sub-population of hepatocytes for which the cell cycle status is unknown.
Not all arrested primary cells, however are permissive to rAAV gene transfer. Bone marrow cells enriched in non cycling CD34+ hematopoietic progenitors will accumulate rAAV DNA, but mostly fail to express the transfered gene (C.Jordan, personal communication). Understanding the determinants of cellular permissivity to AAV will have important implications for the definitions of optimal gene transfer targets in vivo.
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Danos, O. (1998). Adeno-Associated Viral Vectors: Principles and in Vivo Use. In: Merten, OW., Perrin, P., Griffiths, B. (eds) New Developments and New Applications in Animal Cell Technology. Springer, Dordrecht. https://doi.org/10.1007/0-306-46860-3_91
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