Validate genome editing with Xdrop

March 4, 2021
14:00 GMT / 15:00 CET / 9:00 EST

The ease of new genome editing technologies has opened opportunities to explore in more detail the genetic underpinnings of disease, establish more relevant study models, and develop novel advanced therapies. An essential yet often overlooked aspect of that work is validating edits. Learn from Dr. Tom Cunningham how his team humanizes mouse models to understand the genetics of ALS and let Dr. Peter Mouritzen explain how Xdrop® helps ensure edits are happening how and where intended.

PART 1: Genomically Humanised Mouse Models of Neurodegenerative Disease

Thomas Cunningham, Senior Investigator Scientist, MRC

  • Engineering and studying a new generation of knock-in mouse models of motor neuron disease and frontal temporal dementia

  • Genomic humanisation refers to replacement of entire mouse genes with the human orthologous sequence

  • Allele QC is critical in order to accurately study the consequences of pathogenic mutations

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PART 2: CRISPR Validation and Resolving Complex Genomics Landscapes with Xdrop

Dr. Peter Mouritzen, Application Development Services, Samplix, Denmark

  • Indirect Sequence Capture of any genomic region: the Xdrop™ microfluidics system

  • Validation of CRISPR edited cell lines detecting unintended kb-long insertions

  • Identification of integration sites and structural variation in transgenic mice

  • Reconstruction of complex genomic regions: CYP2D6 gene without confounding effects of pseudogenes.

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What you’ll learn from this webinar

  • How current sequencing technologies deal with complex genomic regions
  • An overview of currently available tools for genetic diagnostics and genome validation
  • Genomic approaches capable of coupling target enrichment and long-read sequencing
  • Principles and outcomes of indirect sequence capture and how it works