Personalized medicine

Human induced pluripotent cells (hiPSCs) have been used to study molecular and cellular disease phenotypes as a means to overcome the problems associated with murine, primate, and human embryonic stems cell models in terms of recapitulation of neurodevelopment, neuroanatomy, and genetics as well as ethics. hiPSC technology allows the generation of personalized human neurons from patients.

In contrast to animal models in vitro or in vivo, this technology allows tracking human neurodevelopment using personalized iPSCs from patients under an individual genetic background. Furthermore, powerful genetic engineering tools such as the CRISPR/Cas9 method can be used for disruption, mutation, or deletion of large genomic fragments at a specific locus in the genome of hiPSCs. Importantly, hiPSC technology allows generating customized disease modeling, using direct conversion methods from human somatic cells (e.g. from blood or skin samples) into desired cell types such as neurons using lineage-specific factors, which induce cellular reprogramming. We are currently setting up the hiPSC technology to study the molecular and cellular phenotypes of neurons reprogrammed from hiPSCs derived from autism spectrum disorder patients, Alzheimer’s disease patients, and bipolar disorder patients compared to healthy control subjects. By reversing these identified phenotypes, we aim to develop novel treatments for these conditions.