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Gene Therapy

New Gene Therapy Findings from Kyushu University Described

Published in Gene Therapy Weekly, March 8th, 2012

2012 MAR 8 -- "Retroviral vectors have been employed in clinical trials for gene therapy owing to their relative large packaging capacity, alterable cell tropism, and chromosomal integration for stable transgene expression. However, uncontrollable integrations of transgenes are likely to cause safety issues, such as insertional mutagenesis," scientists in Fukuoka, Japan report.

"A targeted transgene integration system for retroviral vectors, therefore, is a straightforward way to address the insertional mutagenesis issue. Adeno-associated virus (AAV) is the only known virus capable of targeted integration in human cells. In the presence of AAV Rep proteins,...

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