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Researchers at the Salk Institute for Biological Studies are claiming to have achieved a “major milestone” in personalized medicine by proving that a human hair follicle can be converted back into a stem cell and be used, along with gene therapy, to cure disease in a single cell.
The discovery has “catapulted the field of regenerative medicine significantly forward,” according to a Salk news release. Although, several studies have shown that the technology works in mice, the Salk study is the first to show that it can work in human cells.
The discovery was achieved by Juan-Carlos Izpisúa Belmonte, a Salk researcher and director of the Center of Regenerative Medicine in Barcelona, Spain, and Salk molecular biologist Inder Verma. It was published Sunday in the journal Nature.
The researchers focused on Fanconi anemia, a genetic disorder that impairs the body’s ability to fight infection, deliver oxygen, and clot blood. The disease often leads to bone marrow failure, leukemia, and other cancers.
Here is how the Salk release described the procedure, which was done in a Petri dish:
After taking hair or skin cells from patients with Fanconi anemia, the investigators corrected the defective gene in the patients’ cells using gene therapy techniques pioneered in Verma’s laboratory. They then successfully reprogrammed the repaired cells into induced pluripotent stem (iPS) cells using a combination of transcription factors, OCT4, SOX2, KLF4 and cMYC. The resulting FA-iPS cells were indistinguishable from human embryonic stem cells and iPS cells generated from healthy donors.
“We haven’t cured a human being, but we have cured a cell,” Belmonte said in the release. “In theory we could transplant it into a human and cure the disease.”
Clarification: A previous version of this post listed the lead researchers in a different order.