Mouse Models for Drug Discovery. Can New Tools and Technology Improve Translational Power

摘要: The use of mouse models in biomedical research and preclinical drug evaluation is on the rise. advent new molecular genome-altering technologies such as CRISPR/Cas9 allows for genetic mutations to be introduced into germ line a faster less expensively than previous methods. In addition, rapid progress development somatic transgenesis using viral vectors, well manipulations gene expression with siRNAs antisense oligonucleotides, allow even greater exploration genomics systems biology. These technological advances come at time when cost reductions genome sequencing have led identification pathogenic patient populations, providing unprecedented opportunities mice model human disease. ease engineering also offers potential paradigm shift resource sharing speed by which are made available public domain. Predictively, knowledge alone that can quickly remade will provide relief resources encumbered licensing Material Transfer Agreements. For decades, strains provided an exquisite experimental tool study pathophysiology disease assess therapeutic options genetically defined system. However, major limitation has been limited diversity associated common laboratory mice. This overcome recent Collaborative Cross Diversity Outbred tools capable replicating approaching found populations. thus means observe characterize toxicity or efficacy drugs given population. combination traditional contemporary editing tools, along addition modeling systems, synergistic serve make better research, enhancing discovery personalized medicine.