Sa1504 SAFETY PROFILE OF LIVER-DIRECTED, NON-VIRAL GENE DELIVERY BY ENDOSCOPIC-MEDIATED HYDRODYNAMIC INJECTION OF THE BILIARY TRACT IN PIGS

2020 ◽  
Vol 158 (6) ◽  
pp. S-1314
Author(s):  
Yuting Huang ◽  
Robert Kruse ◽  
Thomas Shum ◽  
Hui Ding ◽  
Cem Simsek ◽  
...  
2020 ◽  
Vol 20 ◽  
Author(s):  
L. Hajba ◽  
A. Guttman

: Adeno-associated virus (AAV) is one of the most promising viral gene delivery vectors with long-term gene expression and disease correction featuring high efficiency and excellent safety in human clinical trials. During the production of AAV vectors,there are several quality control (QC)parameters that should be rigorously monitored to comply with clini-cal safety and efficacy. This review gives a short summary of the most frequently used AVV production and purification methods,focusing on the analytical techniques applied to determine the full/empty capsid ratio and the integrity of the encapsidated therapeutic DNA of the products.


2002 ◽  
Vol 73 (2) ◽  
pp. 432-437 ◽  
Author(s):  
Uz Stammberger ◽  
Aima N. Uduehi ◽  
Bartosz Kubisa ◽  
Thierry Roth ◽  
Ralph A. Schmid

Biomaterials ◽  
2013 ◽  
Vol 34 (37) ◽  
pp. 9657-9665 ◽  
Author(s):  
Michael Keeney ◽  
Sheila Onyiah ◽  
Zhe Zhang ◽  
Xinming Tong ◽  
Li-Hsin Han ◽  
...  

Biomaterials ◽  
2011 ◽  
Vol 32 (23) ◽  
pp. 5402-5410 ◽  
Author(s):  
Stephany Y. Tzeng ◽  
Hugo Guerrero-Cázares ◽  
Elliott E. Martinez ◽  
Joel C. Sunshine ◽  
Alfredo Quiñones-Hinojosa ◽  
...  

2013 ◽  
Vol 14 (1) ◽  
pp. 46-60 ◽  
Author(s):  
Weiwei Wang ◽  
Wenzhong Li ◽  
Nan Ma ◽  
Gustav Steinhoff

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