Reprogramming biology: iPSCs and the future of predictive research

Nearly 20 years ago, scientists discovered how to turn back the clock on cellular identity. By introducing a small set of genetic factors critical for cell fate, they reprogrammed adult human cells into a flexible, early developmental state that can self-renew and differentiate into nearly any cell type. These cells, induced pluripotent stem cells (iPSCs), have reshaped how scientists model and study disease.

Unlike embryonic stem cells, which are derived from early‑stage embryos, iPSCs come from adult tissues such as skin or blood. This allows scientists to generate patient‑specific cell types — neurons for studying neurodegenerative diseases, cardiomyocytes for heart conditions, or immune cells for inflammatory disorders.

Because these cells reflect an individual's unique genetic makeup, researchers can use them to study how specific genetic variations contribute to disease, test drug responses across genetic backgrounds and even develop and validate genome-editing strategies.

Researchers at both JAX and NYSCF have advanced protocols to turn these stem cells into key cell types affected in diseases such as Parkinson’s, multiple sclerosis diabetes, and more — accelerating discoveries in drug screening and regenerative medicine.

NYSCF also pioneered the automation of stem-cell production, building one of the world’s largest banks of standardized, patient-derived iPSCs. The Array can generate thousands of high-quality iPSC lines at scale, enabling studies that would be impossible with manual methods.

Yet working with iPSC “lines” (samples of identical cells from single donors) comes with a challenge. Even small differences between these lines, whether from lab processes or inherited from the original donor, can make it hard to compare results across studies. To address this, in 2023 JAX scientists released a reference iPSC line with genomic stability, efficient gene‑editing capacity, and consistent differentiation behavior.

This alliance between JAX and NYSCF paves the way into a new era of biomedical research — one that can help us tackle diseases such as Alzheimer's, cancer and a wide range of rare genetic conditions.

“JAX’s alliance with NYSCF is an exciting step forward in amplifying our impact on human health, building on the bold step we took just over a decade ago in establishing The Jackson Laboratory for Genomic Medicine,” said JAX Chair Emeritus Leo Holt. “Supercharged capabilities in cell models and complementary scientific expertise in areas like cancer and neurodegeneration make me even more hopeful about what JAX will accomplish in the decades to come.”