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Electron Microscopic Evaluation of Vector Quality for Gene Therapy

Published online by Cambridge University Press:  02 July 2020

Qian-Chun Yu ,
Xiaokui Ma ,
Maria Croyle ,
Guang Qu ,
James Huang ,
Guangping Gao ,
Joe Hughes  and
James Wilson
Qian-Chun Yu
Affiliation:
Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA19104
Xiaokui Ma
Affiliation:
Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA19104
Maria Croyle
Affiliation:
Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA19104
Guang Qu
Affiliation:
Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA19104
James Huang
Affiliation:
Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA19104
Guangping Gao
Affiliation:
Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA19104
Joe Hughes
Affiliation:
Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA19104
James Wilson
Affiliation:
Institute for Human Gene Therapy, University of Pennsylvania, Philadelphia, PA19104
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Extract

Successful gene therapy is dependent upon well regulated, long-term expression of transgenes in the target tissue. The development and production of very high quality vectors are one of the most important steps leading to delivery of the desired genes. Although several types of vectors have been used for the delivery of transgene to the target cells of the host, our institute routinely uses recombinant viral vectors including adenovirus and adeno-associated virus (rAAV) for gene therapy studies. To assure high quality of the viral vectors being used for animal experiment as well as clinical trials, a number of quality control protocols, including electron microscopy, was developed and implemented to monitor the overall purity, and density of the vectors.

Transmission electron microscopy (TEM), combined with optimized procedure of negative staining for virus, offers a unique means to generate visual data of the vector preparation; and it has gained increasing application to help assess the quality of vectors.

Type
Biomedical Applications
Information
Microscopy and Microanalysis , Volume 6 , Issue S2: Proceedings: Microscopy & Microanalysis 2000, Microscopy Society of America 58th Annual Meeting, Microbeam Analysis Society 34th Annual Meeting, Microscopical Society of Canada/Societe de Microscopie de Canada 27th Annual Meeting, Philadelphia, Pennsylvania August 13-17, 2000 , August 2000 , pp. 858 - 859
Copyright
Copyright © Microscopy Society of America

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References

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