Success in the pharmaceutical drug development pipeline depends on basic and preclinical research practices that facilitate effective drug discovery and clinical translation. The advent of new molecular genome-altering technologies allows for genetic mutations to be introduced into the germ line of a mouse faster and less expensively than previous methods. In addition, the rapid progress in the development and use of somatic transgenesis using viral vectors, as well as manipulations of gene expression with siRNAs and antisense oligonucleotides (ASO), allow for even greater exploration into genomics and systems biology.
Most standard genetically humanized mouse models only integrate the human coding sequences into the mouse genome. However, recent studies in genomic analysis have revealed the importance of the non-coding genome (both transcribed and non-transcribed), highlighting the need to include non-coding sequences in humanized mouse models. One of the main advantages of Cyagen’s TurboKnockout-Pro approach is that we can introduce larger human DNA fragments (including non-coding sequences) into the mouse genome, so the term "genomically" is used to differentiate these from the regular genetically humanized mouse models.
These technological advances come at a time when cost reductions in genome sequencing have led to the identification of pathogenic mutations in patient populations, providing unprecedented opportunities in the use of mice to model human diseases. With modern advances in genome engineering, one can make precise genomically humanized mouse models faster, more cost-effective, and on many different translationally relevant genetic backgrounds.