Andrew Smith Research Group

Stem cell genome engineering

The stem cell genome engineering group generates genetically manipulated stem cells for use in a wide range of projects at CRM. For example, in order to monitor and investigate the commitment of differentiation of stem cells into more specialised cell types, human embryonic stem cell lines have been generated expressing fluorescent proteins regulated by key genes controlling several important differentiation processes. Another important area of research is to make models of human disease in transgenic animals and human stem cell lines to use as experimental platforms for drug testing, and also to investigate ways for correcting disease mutations in somatic cells.

Dr Andrew Smith

Group Leader

Contact details

Human ES cell line
Human ES cell line with green fluorescent reporter expressed under the control of a gene (GATA6) that indicates cells are being primed to differentiate.

Aims and areas of interest

New technologies are critical to drive forward advances in stem cell and regenerative medicine research. The Stem Cell Genome Engineering Group is focussed on developing innovative genetic engineering technologies to genetically manipulate genes and chromosomes by homologous and site-specific recombination, to underpin studies on fundamental aspects of stem cell biology investigated by many groups at CRM and to generate novel models of human disease. Human disease models in transgenic mice and recapitulation of human diseases using in vitro differentiated genetically modified human ES cells will in combination provide the best experimental approach for investigating cooperating genetic and environmental factors involved in diseases, and eventually facilitate experimental platforms for development of drug therapies. In the future, by making improvements in the efficiency of homologous recombination the aim is to facilitate potential approaches for treating inherited human diseases by gene targeted correction of genetic defects in vivo, or in ex vivo cultured cells that can be used for autologous transplantation.