The scope and technical complexity of assessing the risk of secondary malignancies in gene therapy recipients looms over the US Food and Drug Administration’s focus on the role of insertion site analysis (ISA).
Gene Therapy And Secondary Malignancies: Integration Site Analysis Can Only Be Part Of The Answer
The US FDA seeks to better understand and predict insertional mutagenesis in gene therapies like bluebird bio's Skysona, which use integrating viral vectors, but a workshop on integration site analysis highlighted the approach's complexity and limitations.
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