Sydney West Translational Cancer Research Centre
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Dr Gaetan Burgio: Any Idiot can do it’: An overview of the CRISPR gene editing technology from basic biology to irreproducible research



Children's Medical Research Institute Seminar Room

Dr Gaetan Burgio, Group Leader and Head of the Transgenesis Facility, John Curtain School of Medical Research

Novel precision genetic technologies such as CRISPR/Cas9 genome editing technology offer novel avenues to a better understanding the mechanisms of diseases. Using CRISPR/Cas9 we are able to precisely modify the mouse or the human genome by creating knockout or a specific single nucleotide change to enable the study of the function of the gene of interest. The generation of these models lies on the ability of Cas9 to create a double strand break in the DNA and the repair to occur via the error prone Non-Homologous End Joining (NHEJ) or the precise Homology direct Repair (HDR) mechanisms. A large body of work has been recently dedicated to either improve the technology to generate efficiently knockout or knock-in mouse models (point mutations, tags or floxed alleles) or to cure diseases. The rapid pace of the technology development has generated a lot of excitement but also some disappointment over the lack of reproducibility of the experiments. This led to a substantial loss of research time and funding. This short presentation will give an overview over the basic biology questions to the application of the CRISPR technology to interrogate diseases. I will also discuss the initiatives undertaken to tackle irreproducible research and how to address these problems in the field of gene editing technology.

Host: Leszek Lisowski