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Navigating cell and gene therapy: the benefits of whole-genome profiling


Cell and gene therapy research often requires inserting large, multi-kilobase DNA sequences into the genome. These knock-in experiments enable scientists to investigate gene function and develop new treatments. However, rigorous validation is required to confirm insertion accuracy and detect unintended genomic changes, which remain challenging.

In this webinar, Monica Sentmanat presented long-read sequencing of captured multi-kilobase targets (LOCK-seq). This advanced whole-genome profiling approach precisely confirms multi-kilobase knock-ins and detects genome-wide changes, ensuring reliable research models and enhancing the safety of innovative therapies.

Watch this webinar to learn:

  • How to achieve high on-target coverage in knock-in verification.

  • Strategies for cost-effective LOCK-seq multiplexing and data analysis.

  • The benefits of nanopore sequencing for cell and gene therapy research.

Authors: Monica Sentmanat, Genome Engineering and Stem Cell Center at the McDonnell Genome Institute

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