BIOM30001 Lecture Notes - Lecture 15: Gene Targeting, Gene Knockout, Rabies Virus
13 Sep 2018
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Document Summary
Gene targeting and editing: essential tools for disease modelling. Introduction of a reporter gene: correction of a polymorphism. Tools: cut genomic dna: crispr/cas9, zinc finger nucleases, talens. Result in: gene knockout (ko, promote recombination more efficient gene targeting. Reporters of development can help identify protocols for ipsc differentiation: eg. Example 1: examine da neuron loss in mouse. Reporter genes (3: fluorescent protein weak expression and unpredictable, eg. Very hard to culture neuron in vitro. Example 2: ipsc reprogramming towards interested disease as model cv disease. Patient-derived ipscs are being derived rapidly to model a multitude of specific disease. Single gene disease is easier to examine than polygenetic disease. Remove epigenetic marks by reprogramming reset. Great potential for rare hereditary monogenic diseases. Different backgrounds mask effect of disease-related genes. Gene editing on a common genetic background maybe a better approach. Gwas studies indicate that many diseases su(cid:272)h as alzhei(cid:373)er"s disease are (cid:272)hara(cid:272)terised (cid:271)y.
