Genetic lethality

Airmeith Bioscience has designed controllable and usable models of genetic disorders, where knockout at particular developmental stages is lethal. Recently, we developed a double transgenic model for examination of a gene therapy to treat a specific patient gene mutation in a highly debilitating genetic disorder. The disorder is lethal in mice, making examination of gene therapies in vivo very difficult. First, part of the endogenous mouse gene was replaced with the human sequence containing the specific patient mutation, without disrupting transcriptional or translational signals. By introduction of an antibiotic-controlled cDNA at a separate open locus, protein expression is preserved until antibiotic is removed in a controlled manner to allow disease progression and examination of the gene therapy..