The goal of Kiran Musunuru's lab in Harvard's Department of Stem Cell and Regenerative Biology is to understand the basis for cardiovascular and metabolic human diseases. They do that by studying patients to uncover new gene variants associated with conditions of interest, then studying those variants in model systems: either human cells or mice.
In a recent issue of Cell Stem Cell, Musunuru, Chad Cowan and their colleagues describe a much more efficient tool for doing that disease modeling work in human pluripotent stem cells: a transcription activator-like effector nuclease (TALEN) kit consisting of 834 plasmids. The researchers showed they could use their TALEN kit to quickly and efficiently generate human stem cells edited to carry mutant versions of 15 different disease-associated genes.
Addgene spoke to Musunuru about how the new kit works, the research the kit now makes possible, and how it compares to his CRISPR/Cas9 system.