Unraveling the Function of Disease-Related Gene

695 WordsFeb 24, 20183 Pages
Creating cellular model has been a challenging task of a genetic engineer. However, cellular model became a favorite model for biologists due to its inexpensive in generation and maintenance of culture, avoid risk of losing any precious biological material and expedite the various inherent intriguing biological mechanisms at first instance before testing for the in vivo application. Cellular models also provide a great avenue for studying the disease mechanism and drug discovery. To generate the cellular genetic model, TALENs with the reporters are transfected in human cell line to induce targeted genome modification. Genetically modified mammals can serve as valuable models of human genetic disorders, but the majority of disease-associated alleles represent single-nucleotide replacement that lead to missense, nonsense, and silent mutations (Sauna and Kimchi-Sarfaty, 2011). Compared with other animal models, mice are preferred because of their small body size, their fertilization cycle, and genetically close relationship to human genome. To unravel the function of disease-related genes and to understand the mechanism of disease progression, mouse mutants were generated by pronuclear microinjection of TALEN mRNA targeted to the mouse homologs of human disease genes into one-cell embryos to generate novel Knockout and Knockin mutants. Frontotemporal lobar degeneration (FTLD) is a common cause of presenile dementia caused by the degeneration of neurons in frontal and temporal
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