With CRISPR, Modeling Disease in Mini Organs

By Tanya Lewis

Traditionally, gene therapy efforts have attempted to treat genetic diseases by modifying DNA inside a patient’s body, but it has been a challenge to deliver the genetic material to all the target tissue, let alone to do so safely. But in recent years, advances in gene editing and stem cell research have enabled scientists to correct genetic defects in a patient’s own cells and grow tissue-specific “organoids” in vitro. These mini organs hold promise for modeling disease, screening drugs, and—potentially—replacing defective tissue in patients.

ISCRM’s Benjamin Freedman and his colleagues have developed a way to grow kidney organoids from patient-derived iPSCs, which they use to study diseases like polycystic kidney disease (PKD), which causes balloon-like cysts to grow on kidneys, displacing healthy tissue. His lab uses CRISPR to introduce PKD-associated mutations into kidney cells in vitro, and then grows the cells into kidney organoids that form the cysts characteristic of the disease.

Read the full story at The Scientist.