Based at UQ's Australian Institute for Bioengineering and Nanotechnology (AIBN), the Stem Cell Engineering laboratory lead by Professor Ernst Wolvetang focuses on elucidating and curing neurological diseases that occur in early human development, such as rare childhood leukoencephalopathies, ataxias and Down syndrome, as well as later onset more prevalent diseases, such as and Alzheimers disease. To understand the relationship between gene mutations/duplications and the molecular mechanisms that underlie neuro-pathogenic processes the Wolvetang laboratory uses human induced pluripotent stem cells (iPSC), an artificially created stem cell type established from skin or blood cells donated by patients. Importantly these reprogrammed stem cells capture the genome of the patient, are immortal and can generate all cell types of the human body. Taking advantage of these properties iPSC are next differentiated into various brain cell types in 2D and 3D (brain organoid) culture settings and interrogated at a functional level (activity, neuronal connectivity, myelination, cell migration, beta-amyloid plaque formation etc) and at a genomic level (transcriptome, methylome, proteome) to uncover the gene regulatory networks underlying disease. CRISPR-Cas9 genome editing is subsequently used to either correct disease-causing mutations or introduce these into control iPSC, proving the pathogenicity of particular mutations (monogenic disease) and revealing the inter-relationships between genome alterations that collectively lead to disease (complex disease). Subsequently, these “brain disease in a dish” models are used to screen drugs and test therapeutic modalities on the patients’ own brain cell types (precision medicine) and gene corrected patient iPSC derived cells are also attractive candidates for cellular therapies under development.