Reprogramming mature human cells so they can become any cell type in the body is a key focus of the new Pluripotent Stem Cell Facility at Cincinnati Children’s Hospital Medical Center.
Known as "induced pluripotent stem cells” (iPSCs), they give researchers at Cincinnati Children’s and the UC College of Medicine powerful new tools to study what causes diseases and to grow genetically identical, patient-specific replacement tissues for future therapies, says James Wells, PhD, facility director and associate professor of pediatrics at UC.
"This technology is a bit like the internal combustion engine in terms of how it will drive future advances in stem cell biology,” Wells says. "It allows us to use cells from patients to study what goes wrong at the genetic and cellular level to cause their disease—whether it’s muscular dystrophy, diabetes or any number of degenerative diseases. "This technology could allow us to fix genetic defects and use these cells to generate healthy cells and tissues to treat or cure the patient.”
Pluripotent means the stem cells have the theoretical ability to become any of the more than 200 different cell types found in the human body.
They provide a renewable source of cells for research into the emerging area of regenerative medicine, which focuses on replacement therapies to create living, functional tissues that repair and replace tissues or organ function lost from age, disease, injury or congenital defects.
Because iPSCs come from, and are therefore genetically identical to the patient, they also should be safe in transplantation-based therapies without fear of rejection, Wells says.
The stem cell facility is a central source of human pluripotent stem cell technologies for researchers at Cincinnati Children’s, UC and elsewhere, Wells says.
The facility provides training in the generation and use of pluripotent stem cells. It also is believed to be the only facility of its type in Ohio, Kentucky and Indiana, and one of only a small number in the country.
To generate induced pluripotent stem cells, researchers take skin biopsies from healthy people or patients with specific diseases and grow the skin cells in a petri dish.
Scientists then insert specific genes into a cell’s nucleus, instructing the mature cells to essentially reverse their life cycle and become unspecialized "embryonic-like” cells.
It takes two to three months in a petri dish to make a single batch of iPSCs, says Chris Mayhew, PhD, co-director of the facility.
More information about the facility, including fees, is available at research.cchmc.org/stemcell.