Adenovirus-Adeno-Associated Virus Hybrid for Large-Scale Recombinant Adeno-Associated Virus Production

Recombinant adeno-associated virus (rAAV) holds promise for applications in gene therapy. Advances in clinical studies of rAAV-based gene therapeutics have generated an encouraging momentum in the field of gene therapy; however, one of the major obstacles to the eventual clinical success of rAAV-med...

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Veröffentlicht in:Human gene therapy 2009-09, Vol.20 (9), p.922-929
Hauptverfasser: HONGWEI ZHANG, JUN XIE, QING XIE, WILSON, James M, GUANGPING GAO
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container_end_page 929
container_issue 9
container_start_page 922
container_title Human gene therapy
container_volume 20
creator HONGWEI ZHANG
JUN XIE
QING XIE
WILSON, James M
GUANGPING GAO
description Recombinant adeno-associated virus (rAAV) holds promise for applications in gene therapy. Advances in clinical studies of rAAV-based gene therapeutics have generated an encouraging momentum in the field of gene therapy; however, one of the major obstacles to the eventual clinical success of rAAV-mediated gene therapy is the need for large-scale production of clinical-grade vectors. The transfection-based rAAV production method is well suited for preclinical studies in small animal models, but it is difficult to support large-scale clinical studies with this method. In the past decade, several scalable rAAV production methods have emerged from extensive efforts to develop large-scale manufacturing processes. Among those, the recombinant adenovirus-AAV infection method has some unique features in vector quality and yield. This minireview provides an overview of this scaleable rAAV production platform, describing its basic components and biological mechanisms and process.
doi_str_mv 10.1089/hum.2009.125
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Psychology ; Gene therapy ; Genetic aspects ; Genetic Therapy - methods ; Genetic Vectors ; Health aspects ; Health. Pharmaceutical industry ; HeLa Cells ; Humans ; Industrial applications and implications. Economical aspects ; Medical sciences ; Production processes ; Recombination, Genetic ; Transfusions. Complications. Transfusion reactions. 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Advances in clinical studies of rAAV-based gene therapeutics have generated an encouraging momentum in the field of gene therapy; however, one of the major obstacles to the eventual clinical success of rAAV-mediated gene therapy is the need for large-scale production of clinical-grade vectors. The transfection-based rAAV production method is well suited for preclinical studies in small animal models, but it is difficult to support large-scale clinical studies with this method. In the past decade, several scalable rAAV production methods have emerged from extensive efforts to develop large-scale manufacturing processes. Among those, the recombinant adenovirus-AAV infection method has some unique features in vector quality and yield. This minireview provides an overview of this scaleable rAAV production platform, describing its basic components and biological mechanisms and process.</description><subject>Adeno-associated virus</subject><subject>Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy</subject><subject>Animal models in research</subject><subject>Animals</subject><subject>Applied cell therapy and gene therapy</subject><subject>Biological and medical sciences</subject><subject>Biotechnology</subject><subject>Biotechnology - methods</subject><subject>Cell Line</subject><subject>Cercopithecus aethiops</subject><subject>Dependovirus - genetics</subject><subject>Dependovirus - metabolism</subject><subject>Dependovirus - physiology</subject><subject>Dependoviruses</subject><subject>Fundamental and applied biological sciences. Psychology</subject><subject>Gene therapy</subject><subject>Genetic aspects</subject><subject>Genetic Therapy - methods</subject><subject>Genetic Vectors</subject><subject>Health aspects</subject><subject>Health. Pharmaceutical industry</subject><subject>HeLa Cells</subject><subject>Humans</subject><subject>Industrial applications and implications. 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Economical aspects</topic><topic>Medical sciences</topic><topic>Production processes</topic><topic>Recombination, Genetic</topic><topic>Transfusions. Complications. Transfusion reactions. 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subjects Adeno-associated virus
Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy
Animal models in research
Animals
Applied cell therapy and gene therapy
Biological and medical sciences
Biotechnology
Biotechnology - methods
Cell Line
Cercopithecus aethiops
Dependovirus - genetics
Dependovirus - metabolism
Dependovirus - physiology
Dependoviruses
Fundamental and applied biological sciences. Psychology
Gene therapy
Genetic aspects
Genetic Therapy - methods
Genetic Vectors
Health aspects
Health. Pharmaceutical industry
HeLa Cells
Humans
Industrial applications and implications. Economical aspects
Medical sciences
Production processes
Recombination, Genetic
Transfusions. Complications. Transfusion reactions. Cell and gene therapy
Vero Cells
title Adenovirus-Adeno-Associated Virus Hybrid for Large-Scale Recombinant Adeno-Associated Virus Production
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