Leveraging quadplexed digital PCR to characterize gene therapy vectors

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Leveraging quadplexed digital PCR to characterize gene therapy vectors

Authors

Tereshko, L. R.; Ryals, M.; Gagnon, J.; Admanit, R.; Mason, C.

Abstract

Currently there is a lack of high-throughput, low material-input methods to screen early-stage product quality of viral and non-viral gene therapy products. Here we propose using multiplex droplet digital PCR (ddPCR) to screen and characterize vector sequences. We describe the adaptation of a Poisson-multinomial model to quantitate integrity of any combination of 4 targets in multiplexed ddPCR. We show the success and limitations of model employment and provide some suggested best practices.

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