Novel gene transfer methods
Scientists are exploring novel gene transfer methods that may help overcome some of the challenges associated with the current CAR T products. At present, viral vectors (lentiviruses or retroviruses) are used to genetically engineer CAR T cells. A DNA vector (i.e., a transposon) may provide an alternative.
Nobuhiro Nishio (Japan) explained that transposons are faster, less expensive, and safer than viral vectors. However, the main disadvantage of transposons is the reduced gene transduction efficiency. Nishio’s laboratory has managed to improve this efficiency for the so-called PiggyBac transposon system. They have started a clinical trial with CD19-directed CAR T cell therapy in ALL. Other speakers (including Michael Hudecek, Sarah Tettamanti, and Friederike Schmitt) discussed virus-free gene transfer using the Sleeping Beauty transposon platform.
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