To create a gene knock-in to generate transgenics that ectopically express a mutant protein, then gene targeting mouse embryonic stem (ES) cells is typically performed. As proteins with a human amino acid sequence can have different biochemical characteristics from their mouse orthologs, transgenics have often been made with human cDNAs, and targeting has involved placing human coding sequences into the orthologous mouse gene. This strategy using coding sequences can result in a more accurate mouse disease model than working with a mutant mouse protein.
Genomically humanized mice can also be achieved by bacterial artificial chromosome (BAC) transgenesis, inserting an entire human gene, including its regulatory elements, into the mouse genome. Cyagen’s TurboKnockout® platform combined with BAC technology brings genomically humanized mouse models - on an accelerated timeline. Inserting the entire human gene has increasingly shown improved success of translational research.
Compared with CRISPR-Pro-based techniques, TurboKnockout® is free of patent disputes, provides precise models on a comparable timeline, and is the technology of choice among our clients performing drug development projects.
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