TY - JOUR
T1 - Adeno-associated virus vector-mediated transgene integration into neurons and other nondividing cell targets
AU - Wu, Ping
AU - Phillips, M. Ian
AU - Bui, John
AU - Terwilliger, Ernest F.
PY - 1998/7
Y1 - 1998/7
N2 - The site-specific integration of wild-type adeno-associated virus (wtAAV) into the human genome is a very attractive feature for the development of AAV-based gene therapy vectors. However, knowledge about integration of wtAAV, as well as currently configured recombinant AAV (rAAV) vectors, is limited. By using a modified Alu-PCR technique to amplify and sequence the vector-cellular junctions, we provide the first direct evidence both in vitro and in vivo of rAAV-mediated transgene integration in several types of nondividing cells, including neurons. This novel technique will be highly useful for further delineating the mechanisms underlying AAV-mediated integration, including issues of frequency, site preference, and DNA rearrangement in human as well as animal cells. Results from these studies should be beneficial for the development of the next generation of gene delivery vectors.
AB - The site-specific integration of wild-type adeno-associated virus (wtAAV) into the human genome is a very attractive feature for the development of AAV-based gene therapy vectors. However, knowledge about integration of wtAAV, as well as currently configured recombinant AAV (rAAV) vectors, is limited. By using a modified Alu-PCR technique to amplify and sequence the vector-cellular junctions, we provide the first direct evidence both in vitro and in vivo of rAAV-mediated transgene integration in several types of nondividing cells, including neurons. This novel technique will be highly useful for further delineating the mechanisms underlying AAV-mediated integration, including issues of frequency, site preference, and DNA rearrangement in human as well as animal cells. Results from these studies should be beneficial for the development of the next generation of gene delivery vectors.
UR - http://www.scopus.com/inward/record.url?scp=0031779697&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=0031779697&partnerID=8YFLogxK
U2 - 10.1128/jvi.72.7.5919-5926.1998
DO - 10.1128/jvi.72.7.5919-5926.1998
M3 - Article
C2 - 9621054
AN - SCOPUS:0031779697
SN - 0022-538X
VL - 72
SP - 5919
EP - 5926
JO - Journal of virology
JF - Journal of virology
IS - 7
ER -