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 vector-mediated gene delivery to the vasculature and kidney.

2005 ◽  
Vol 52 (2) ◽  
pp. 293-299 ◽  
Author(s):  
Matthias H Kapturczak ◽  
Sifeng Chen ◽  
Anupam Agarwal
Keyword(s):  
Gene Delivery ◽  
Transgene Expression ◽  
Vascular Diseases ◽  
Viral Gene ◽  
Therapeutic Approaches ◽  
Adeno Associated Virus ◽  
Coding Sequences ◽  
Transgene Delivery ◽  
Viral Gene Delivery

Relatively successful elsewhere, gene delivery aimed at the vasculature and kidney has made very little progress. In the kidney, the hurdles are related to the unique structure-function relationships of this organ and in the blood vessels to a variety of, mostly endothelial, factors making the delivery of transgenes very difficult. Among gene-therapeutic approaches, most viral gene delivery systems utilized to date have shown significant practical and safety-related limitations due to the level and duration of recombinant transgene expression as well as their induction of a significant host immune response to vector proteins. Recombinant adeno-associated virus (rAAV) vectors appear to offer a vehicle for safe, long-term transgene expression. rAAV-based vectors are characterized by a relative non-immunogenicity and the absence of viral coding sequences. Furthermore, they allow for establishment of long-term latency without deleterious effects on the host cell. This brief review addresses problems related to transgene-delivery to kidney and vasculature with particular attention given to rAAV vectors. The potential for gene therapy as a strategy for selected renal and vascular diseases is also discussed.

scholarly journals Cationic lipids with a cyclen headgroup: synthesis and structure–activity relationship studies as non-viral gene vectors

RSC Advances ◽  
2017 ◽  
Vol 7 (30) ◽  
pp. 18681-18689 ◽  
Author(s):  
De-Chun Chang ◽  
Yi-Mei Zhang ◽  
Ji Zhang ◽  
Yan-Hong Liu ◽  
Xiao-Qi Yu
Keyword(s):  
Gene Delivery ◽  
Structure Activity Relationship ◽  
Cationic Lipids ◽  
Activity Relationship ◽  
Viral Gene ◽  
Structure Activity Relationships ◽  
Structure Activity ◽  
Gene Delivery Vectors ◽  
Gene Vectors ◽  
Viral Gene Delivery

The structure–activity relationships of cyclen-based cationic lipids as non-viral gene delivery vectors were studied and clarified.

Cholesterol derived cationic lipids as potential non-viral gene delivery vectors and their serum compatibility

2016 ◽  
Vol 26 (10) ◽  
pp. 2401-2407 ◽  
Author(s):  
Jia Ju ◽  
Meng-Lei Huan ◽  
Ning Wan ◽  
Yi-Lin Hou ◽  
Xi-Xi Ma ◽  
...  
Keyword(s):  
Gene Delivery ◽  
Cationic Lipids ◽  
Viral Gene ◽  
Gene Delivery Vectors ◽  
Viral Gene Delivery

Biotinylated Cyclen-Contained Cationic Lipids as Non-Viral Gene Delivery Vectors

2013 ◽  
Vol 82 (4) ◽  
pp. 376-383 ◽  
Author(s):  
Qiang Liu ◽  
Wen-Jing Yi ◽  
Yi-Mei Zhang ◽  
Ji Zhang ◽  
Liandi Guo ◽  
...  
Keyword(s):  
Gene Delivery ◽  
Cationic Lipids ◽  
Viral Gene ◽  
Gene Delivery Vectors ◽  
Viral Gene Delivery

RGD peptide-based non-viral gene delivery vectors targeting integrin αvβ3 for cancer therapy

2018 ◽  
Vol 27 (1) ◽  
pp. 1-11 ◽  
Author(s):  
Shuang Fu ◽  
Xiaodong Xu ◽  
Yu Ma ◽  
Shubiao Zhang ◽  
Shufen Zhang
Keyword(s):  
Cancer Therapy ◽  
Gene Delivery ◽  
Integrin Αvβ3 ◽  
Rgd Peptide ◽  
Viral Gene ◽  
Gene Delivery Vectors ◽  
Viral Gene Delivery
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