Dept. of Life Science Frontiers
Knut Woltjen (Associate Professor)
Knut Woltjen Ph.D.
iPS cell technology is revolutionizing personalized medicine, allowing us to safely study patient genomes by proxy. Employing reprogramming and genome engineering technologies, the Woltjen Lab ultimately aims to raise understanding of human disease predisposition and development through functional genomics.
Genome engineering plays a major role in human disease research. Our lab was the first in CiRA to generate knockout human iPS cells using designer nucleases (Sakuma et al., 2013). We routinely use CRISPR/Cas9 to target reporter genes to the AAVS1 locus in order to achieve stable expression with the intention of tracking differentiated cells in vitro and in vivo (Oceguera et al., 2015).
Reprogramming mechanisms remain obscure. Continuing to use the piggyBac (PB) transposon as a versatile and excisable reprogramming vector (Woltjen et al., 2009), we are studying the importance of factor stoichiometry on reprogramming outcomes. Our recent studies revealed a fundamental difference in cloned Klf4 that affects absolute levels of KLF4 protein, influencing both the initiation and stabilization of true iPS cells (Kim et al., 2015).