Disease in a dish: Emory establishes iPSC laboratory

By Quinn Eastman | Woodruff Health Sciences Center | Nov. 28, 2012

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The Laboratory of Translational Cell Biology will specialize in neurological disorders such as fragile X syndrome, autism, spinal muscular atrophy, Alzheimer’s disease and amyotrophic lateral sclerosis.

Recent advances in induced pluripotent stem cell (iPSC) technology allow researchers to take a patient's skin or blood cells, reprogram them into an immature, embryonic-like state, and then differentiate them into particular tissues. This technology could open doors for truly personalized medicine and drug discovery.

Shinya Yamanaka and John Gurdon were awarded the 2012 Nobel Prize in Medicine for the discovery that differentiated cells in the body can be reprogrammed. These findings have opened up new possibilities for what is called "disease in a dish" — using human cells to establish a patient-specific model of disease. This makes it possible to directly test drugs on the patient's cells, which have been reprogrammed and differentiated into the specific cell type that is most affected by the disease. This approach could be used for drug discovery, and ultimately to inform physicians on the best means to treat the patient.

Laboratory for generation, utilization of iPSCs established at Emory

Emory University School of Medicine, through the Office of the Executive Dean for Research, has established a laboratory dedicated to the generation and utilization of iPSCs for translational research and drug screening.

The Laboratory of Translational Cell Biology (LTCB) will specialize in neurological disorders such as:

  • fragile X syndrome
  • autism
  • spinal muscular atrophy
  • Alzheimer's disease
  • amyotrophic lateral sclerosis.
The LTCB is also conducting research on the use of iPSCs to study genetic defects that cause infertility. A particular area of interest is diseases with impairments in cell structure and dynamics, which need the use of microscope-based assays to visualize disease processes and identify how drugs may restore cell function.

The LTCB will develop iPSCs as tools for research and drug screening, rather than introducing iPSCs into patients as therapies. In addition, the culture of iPSCs does not involve the destruction or manipulation of human embryos.

The Scientific Director of the LTCB is Gary Bassell, professor of cell biology and neurology at Emory University School of Medicine. The associate director is Wilfried Rossoll, assistant professor of cell biology.

"iPSC technology will be especially helpful in situations where an animal model is not available or does not accurately reproduce aspects of a disease," Rossoll says. "In addition, it could allow us to establish in vitro models for human neurologic disease, since it's not possible to take biopsy material from the spinal cord or the brain."

A new faculty member in the Department of Cell Biology has joined the LTCB: Charles Easley, a stem cell biologist. While working at the University of Pittsburgh with Gerald Schatten, Easley was able to coax iPSCs into becoming sperm precursor cells. The results were recently published in the journal Cell Reports. Easley has expertise to derive and characterize human iPSCs, and will play an important leadership role to facilitate collaborations between the LTCB with Emory faculty.

Bassell, Rossoll and Easley say they will plan activities and partnerships for the LTCB such as: