Directed evolution of adeno-associated virus yields enhanced gene delivery vectors

2006 ◽  
Vol 24 (2) ◽  
pp. 198-204 ◽  
Author(s):  
Narendra Maheshri ◽  
James T Koerber ◽  
Brian K Kaspar ◽  
David V Schaffer
Keyword(s):  
Gene Delivery ◽  
Directed Evolution ◽  
Adeno Associated Virus ◽  
Gene Delivery Vectors

Recent advances in the analysis full/empty capsid ratio and genome integrity of adeno-associated virus (AAV) gene delivery vectors

2020 ◽  
Vol 20 ◽  
Author(s):  
L. Hajba ◽  
A. Guttman
Keyword(s):  
Gene Delivery ◽  
High Efficiency ◽  
Analytical Techniques ◽  
Viral Gene ◽  
Adeno Associated Virus ◽  
Gene Delivery Vectors ◽  
Empty Capsid ◽  
Purification Methods ◽  
Viral Gene Delivery

: 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.

scholarly journals Adeno-Associated Virus (AAV) Capsid Stability and Liposome Remodeling During Endo/Lysosomal pH Trafficking

Viruses ◽  
2020 ◽  
Vol 12 (6) ◽  
pp. 668 ◽  
Author(s):  
Bridget Lins-Austin ◽  
Saajan Patel ◽  
Mario Mietzsch ◽  
Dewey Brooke ◽  
Antonette Bennett ◽  
...  
Keyword(s):  
Gene Delivery ◽  
Adeno Associated Virus ◽  
Lysosomal Ph ◽  
Negative Stain ◽  
Gene Delivery Vectors ◽  
Successful Infection ◽  
Monogenic Diseases ◽  
The Stability ◽  
Ssdna Viruses ◽  
Capsid Stability

Adeno-associated viruses (AAVs) are small, non-pathogenic ssDNA viruses being used as therapeutic gene delivery vectors for the treatment of a variety of monogenic diseases. An obstacle to successful gene delivery is inefficient capsid trafficking through the endo/lysosomal pathway. This study aimed to characterize the AAV capsid stability and dynamics associated with this process for a select number of AAV serotypes, AAV1, AAV2, AAV5, and AAV8, at pHs representative of the early and late endosome, and the lysosome (6.0, 5.5, and 4.0, respectively). All AAV serotypes displayed thermal melt temperatures that varied with pH. The stability of AAV1, AAV2, and AAV8 increased in response to acidic conditions and then decreased at pH 4.0. In contrast, AAV5 demonstrated a consistent decrease in thermostability in response to acidification. Negative-stain EM visualization of liposomes in the presence of capsids at pH 5.5 or when heat shocked showed induced remodeling consistent with the externalization of the PLA2 domain of VP1u. These observations provide clues to the AAV capsid dynamics that facilitate successful infection. Finally, transduction assays revealed a pH and temperature dependence with low acidity and temperatures > 4 °C as detrimental factors.

scholarly journals Directed Evolution of Adeno-associated Virus for Enhanced Gene Delivery and Gene Targeting in Human Pluripotent Stem Cells

2012 ◽  
Vol 20 (2) ◽  
pp. 329-338 ◽  
Author(s):  
Prashanth Asuri ◽  
Melissa A Bartel ◽  
Tandis Vazin ◽  
Jae-Hyung Jang ◽  
Tiffany B Wong ◽  
...  
Keyword(s):  
Stem Cells ◽  
Gene Delivery ◽  
Directed Evolution ◽  
Gene Targeting ◽  
Pluripotent Stem Cells ◽  
Human Pluripotent Stem Cells ◽  
Adeno Associated Virus

In Vivo-Directed Evolution of a New Adeno-Associated Virus for Therapeutic Outer Retinal Gene Delivery from the Vitreous

2013 ◽  
Vol 5 (189) ◽  
pp. 189ra76-189ra76 ◽  
Author(s):  
D. Dalkara ◽  
L. C. Byrne ◽  
R. R. Klimczak ◽  
M. Visel ◽  
L. Yin ◽  
...  
Keyword(s):  
Gene Delivery ◽  
Directed Evolution ◽  
Adeno Associated Virus
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