Product-Related Impurities in Clinical-Grade Recombinant AAV Vectors: Characterization and Risk Assessment
نویسندگان
چکیده
منابع مشابه
Product-Related Impurities in Clinical-Grade Recombinant AAV Vectors: Characterization and Risk Assessment
Adeno-associated virus (AAV)-based vectors expressing therapeutic genes continue to demonstrate great promise for the treatment of a wide variety of diseases and together with other gene transfer vectors represent an emerging new therapeutic paradigm comparable in potential impact on human health to that achieved by recombinant proteins and vaccines. A challenge for the current pipeline of AAV-...
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NOVEMBER 2005 Proteins can self-associate. For some proteins, self-association is natural and required for their function (e.g., tumor necrosis factor, TNF, is a homotrimer). However, proteins isolated from their native environment (such as in a vial or in an in-process fluid rather than in a cell or in plasma) can selfassociate into nonnative oligomers, both covalent and noncovalent. This has ...
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Adeno-associated virus (AAV) has become a vector of choice for the treatment of a variety of genetic diseases that require safe and long-term delivery of a missing protein. Muscle-directed gene transfer for delivery of protective antibodies against AIDS viruses and other pathogens has been used experimentally in mice and monkeys. Here we examined a number of variations to AAV vector design for ...
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Transitioning to human trials from pre-clinical models resulted in the emergence of inhibitory AAV vector immune responses which has become a hurdle for sustained correction. Early animal studies did not predict the full range of host immunity to the AAV vector in human studies. While pre-existing antibody titers against AAV vectors has been a lingering concern, cytotoxic T-cell (CTL) responses...
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Adeno-associated virus (AAV) vectors have emerged as a safe and efficient gene therapy platform. One complication is that a significant amount of empty particles have always been generated as impurities during AAV vector production. However, the effects of such particles on AAV vector performance remain unclear. Here we systemically evaluated the biological properties of three types of "empty" ...
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ژورنال
عنوان ژورنال: Biomedicines
سال: 2014
ISSN: 2227-9059
DOI: 10.3390/biomedicines2010080