scholarly journals Long-term systemic therapy of Fabry disease in a knockout mouse by adeno-associated virus-mediated muscle-directed gene transfer

2002 ◽  
Vol 99 (21) ◽  
pp. 13777-13782 ◽  
Author(s):  
H. Takahashi ◽  
Y. Hirai ◽  
M. Migita ◽  
Y. Seino ◽  
Y. Fukuda ◽  
...  
1996 ◽  
Vol 62 (6) ◽  
pp. 1669-1676 ◽  
Author(s):  
Michael G Kaplitt ◽  
Xiao Xiao ◽  
Richard J Samulski ◽  
Juan Li ◽  
Kaie Ojamaa ◽  
...  

2003 ◽  
Vol 7 (5) ◽  
pp. 580-587 ◽  
Author(s):  
Reuben Matalon ◽  
Sankar Surendran ◽  
Peter L Rady ◽  
Michael J Quast ◽  
Gerald A Campbell ◽  
...  

1999 ◽  
Vol 380 (6) ◽  
Author(s):  
H. Büeler

AbstractAdeno-associated virus (AAV) is a defective, non-pathogenic human parvovirus that depends for growth on coinfection with a helper adenovirus or herpes virus. Recombinant adeno-associated viruses (rAAVs) have attracted considerable interest as vectors for gene therapy. In contrast to other gene delivery systems, rAAVs lack all viral genes and show long-term gene expression


2004 ◽  
Vol 56 (2) ◽  
pp. 278-284 ◽  
Author(s):  
Hyun-Jeong Oh ◽  
Eun-Sook Park ◽  
Seongman Kang ◽  
Inho Jo ◽  
Sung-Chul Jung

Gene Therapy ◽  
2003 ◽  
Vol 10 (21) ◽  
pp. 1807-1813 ◽  
Author(s):  
J Li ◽  
D Wang ◽  
S Qian ◽  
Z Chen ◽  
T Zhu ◽  
...  

2014 ◽  
Vol 25 (6) ◽  
pp. 517-528 ◽  
Author(s):  
Marianne S. Carlon ◽  
Dragana Vidović ◽  
James Dooley ◽  
Marina Mori da Cunha ◽  
Michael Maris ◽  
...  

2000 ◽  
Vol 2 (2) ◽  
pp. 147-152 ◽  
Author(s):  
Shohei Watanabe ◽  
Tomoyuki Imagawa ◽  
Gregory P. Boivin ◽  
Guangping Gao ◽  
James M. Wilson ◽  
...  

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