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Gene-editing evolved – combining CRISPR, ZFNs and rAAV for maximum versatility and minimal hassle

Date: October 21st, 2014 16: 00-16: 30 GMT (London time)

Genomic advances in preceding decades have led to a wealth of information on the genetic basis of disease. One way to leverage this information is through development of in vitro cell line models in which patient-relevant mutations are precisely recapitulated, allowing novel drug target identification and validation, and the realisation of personalised therapies. In this webinar we discuss Horizon’s gene-editing platform GENESIS and our custom cell line development service, including selecting the right tool for the right targeting outcome, systematic optimisation of the GENESIS platform, combining CRISPR, ZFNs and rAAV technologies to improve targeting efficiency, and advanced experimental design for knock-ins.

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The Clinical Application, Development and Validation of Cell-free DNA Assays/Platforms

Date: October 23rd, 2014 16: 00-16: 40 GMT (London time)

Molecular characterisation of circulating tumor cells (CTCs) and/or cell free tumor DNA (cfDNA) offers the opportunity for personalized targeted therapy and real-time monitoring of treatment efficacy. The development of novel platforms to assess the presence of cfDNA is challenging due to the difficulty in obtaining a sustainable source of patient-relevant material. This study presents the design and validation of our HDx Multiplex Low Copy Number Reference Standards and their performance in assays optimised for cfDNA detection. The results indicate the successful generation of precisely defined reference material including one EGFR standard containing 25 copies of each G719S, ΔE746-A750, T790M, L858R and L861Q mutation in a total EGFR background of 50000. This equates to 1 mutant cell in 1000 wild type cells and an appropriate level to analyse the performance of the cfDNA assays. We will discuss the clinical application, development and validation of reference materials for cell free DNA assays

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