Research Overview

Thirty years after the Human Genome Project began, we now live in an era of widespread human genome sequencing. Yet, most functions encoded in the human genome remain mysterious. Human induced pluripotent stem (iPS) cells maintain the same genome as their donor and gain the capacity to differentiate into cell types affected by genetic disease. Therefore, iPSCs represent a powerful approach in functional genomics and personalized medicine.

The Woltjen Lab develops and deploys cutting-edge gene-editing technologies in iPSCs to study and gain "total control of the human genome". Our goal is to understand the genome and master techniques required to correct mutations and append new gene functions. Towards this goal, the Woltjen Lab developed methods to precisely edit single-nucleotides and create deletions, the two most common classes of pathogenic human genetic variants. Furthermore, we are establishing precise editing of repetitive DNA sequences which are highly variable throughout human evolution and amongst individuals. Our research addresses significant technical hurdles towards understanding genome function.