scholarly journals Gene therapy and viral vectors. Adeno-associated virus vectors and gene therapy.

Uirusu ◽  
1997 ◽  
Vol 47 (2) ◽  
pp. 221-230
Author(s):  
Masashi Urabe ◽  
Keiya Ozawa
Keyword(s):  
Gene Therapy ◽  
Viral Vectors ◽  
Adeno Associated Virus ◽  
Virus Vectors

Adeno-associated virus vectors for human gene therapy

Gene Therapy ◽  
2020 ◽  
pp. 77-92
Author(s):  
Jeffrey S. Bartlett ◽  
Richard J. Samulski
Keyword(s):  
Gene Therapy ◽  
Human Gene ◽  
Adeno Associated Virus ◽  
Human Gene Therapy ◽  
Virus Vectors

Capsid-Engineering for Central Nervous System-Directed Gene Therapy with Adeno-Associated Virus Vectors

2021 ◽  
Vol 32 (19-20) ◽  
pp. 1096-1119
Author(s):  
Josephine Macdonald ◽  
Jennifer Marx ◽  
Hildegard Büning
Keyword(s):  
Gene Therapy ◽  
Central Nervous System ◽  
Nervous System ◽  
Adeno Associated Virus ◽  
Virus Vectors

Engineering adeno-associated virus vectors for gene therapy

2020 ◽  
Vol 21 (4) ◽  
pp. 255-272 ◽  
Author(s):  
Chengwen Li ◽  
R. Jude Samulski
Keyword(s):  
Gene Therapy ◽  
Adeno Associated Virus ◽  
Virus Vectors

Adeno-Associated Viral Vectors for Gene Transfer and Gene Therapy

1999 ◽  
Vol 380 (6) ◽  
Author(s):  
H. Büeler
Keyword(s):  
Gene Expression ◽  
Gene Therapy ◽  
Gene Transfer ◽  
Gene Delivery ◽  
Viral Vectors ◽  
Delivery Systems ◽  
Adeno Associated Virus ◽  
Viral Genes ◽  
Virus Recombinant

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

Immunogenic Issues Concerning Recombinant Adeno-Associated Virus Vectors for Gene Therapy

2002 ◽  
Vol 2 (4) ◽  
pp. 485-500 ◽  
Author(s):  
J. Sun ◽  
S. Chatterjee ◽  
K. Wong, Jr.
Keyword(s):  
Gene Therapy ◽  
Adeno Associated Virus ◽  
Virus Vectors

scholarly journals Establishment of a Novel Cell Line for the Enhanced Production of Recombinant Adeno-Associated Virus Vectors for Gene Therapy

2014 ◽  
Vol 25 (11) ◽  
pp. 929-941 ◽  
Author(s):  
Stifani Satkunanathan ◽  
Jun Wheeler ◽  
Robin Thorpe ◽  
Yuan Zhao
Keyword(s):  
Gene Therapy ◽  
Cell Line ◽  
Adeno Associated Virus ◽  
Virus Vectors ◽  
Enhanced Production

scholarly journals Weighing the DNA content of Adeno-Associated Virus vectors with zeptogram precision using nanomechanical resonators

2021 ◽  
Author(s):  
Georgios Katsikis ◽  
Iris E Hwang ◽  
Wade Wang ◽  
Vikas S Bhat ◽  
Nicole L McIntosh ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Quality Control ◽  
Viral Vectors ◽  
Analytical Ultracentrifugation ◽  
Vaccine Development ◽  
Turnaround Time ◽  
Charge Detection ◽  
Adeno Associated Virus ◽  
Nanomechanical Resonators

Quantifying the composition of viral vectors used in vaccine development and gene therapy is critical for assessing their functionality. Adeno-Associated Virus (AAV) vectors, which are the most widely used viral vectors for in-vivo gene therapy, are typically characterized using PCR, ELISA, and Analytical Ultracentrifugation which require laborious protocols or hours of turnaround time. Emerging methods such as Charge-Detection Mass Spectroscopy, Static Light Scattering, and Mass Photometry offer turnaround times of minutes for measuring AAV mass, but mostly require purified AAV-based reference materials for calibration. Here, we demonstrate a method for using Suspended Nanomechanical Resonators (SNR) to directly measure both AAV mass and aggregation from a few microliters of sample within minutes. We achieve a resolution near 10 zeptograms which corresponds to 1% of the genome holding capacity of the AAV capsid. Our results show the potential of our method for providing real-time quality control of viral vectors during biomanufacturing.

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