News Column

Studies from University of Florida Yield New Information about Gene Therapy

June 26, 2014

By a News Reporter-Staff News Editor at Gene Therapy Weekly -- Fresh data on Biotechnology are presented in a new report. According to news reporting from Gainesville, Florida, by NewsRx journalists, research stated, "Scalable and genetically stable recombinant adeno-associated virus (rAAV) production systems combined with facile adaptability for an extended repertoire of AAV serotypes are required to keep pace with the rapidly increasing clinical demand."

The news correspondents obtained a quote from the research from the University of Florida, "For scalable high-titer production of the full range of rAAV serotypes 1-12, we developed OneBac, consisting of stable insect Sf9 cell lines harboring silent copies of AAV1-12 rep and cap genes induced upon infection with a single baculovirus that also carries the rAAV genome. rAAV burst sizes reach up to 5x10(5) benzonase-resistant, highly infectious genomic particles per cell, exceeding typical yields of current rAAV production systems. In contrast to recombinant rep/cap baculovirus strains currently employed for large-scale rAAV production, the Sf9rep/cap cell lines are genetically stable, leading to undiminished rAAV burst sizes over serial passages."

According to the news reporters, the research concluded: "Thus, OneBac combines full AAV serotype options with the capacity for stable scale-up production, the current bottleneck for the transition of AAV from gene therapy trials to routine clinical treatment."

For more information on this research see: OneBac: Platform for Scalable and High-Titer Production of Adeno-Associated Virus Serotype 1-12 Vectors for Gene Therapy. Human Gene Therapy, 2014;25(3):212-222. Human Gene Therapy can be contacted at: Mary Ann Liebert, Inc, 140 Huguenot Street, 3RD Fl, New Rochelle, NY 10801, USA. (Mary Ann Liebert, Inc. -; Human Gene Therapy -

Our news journalists report that additional information may be obtained by contacting M. Mietzsch, University of Florida, Coll Med, Dept. of Pediat, Gainesville, FL 32610, United States. Additional authors for this research include S. Grasse, C. Zurawski, S. Weger, A. Bennett, M. Agbandje-McKenna, N. Muzyczka, S. Zolotukhin and R. Heilbronn (see also Biotechnology).

Keywords for this news article include: Biotechnology, Florida, Therapy, Gainesville, United States, North and Central America

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Source: Gene Therapy Weekly

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