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Researchers Create First Stem Cells Using CRISPR Genome Activation

Dr. Sheng Ding
Sheng Ding and his team discovered that activating a single gene is sufficient to change skin cells into stem cells. [Photo: Gladstone Institutes]


“This is a new way to make induced pluripotent stem cells that is fundamentally different from how they’ve been created before,” said author Sheng Ding, PhD, a senior investigator at Gladstone. “At the beginning of the study, we didn’t think this would work, but we wanted to at least try to answer the question: can you reprogram a cell just by unlocking a specific location of the genome? And the answer is yes.”

Pluripotent stem cells can be turned into virtually any cell type in the body. As a result, they are a key therapeutic resource for currently incurable conditions, such as heart failure, Parkinson’s disease, and blindness. They also provide excellent models to study diseases and important tools to test new drugs in human cells.

In 2006, Gladstone Senior Investigator Shinya Yamanaka, MD, PhD, discovered he could make stem cells—dubbed induced pluripotent stem cells (iPSCs)—by treating ordinary skin cells with four key proteins. These proteins, called transcription factors, work by changing which genes are expressed in the cell, turning off genes associated with skin cells, and turning on genes associated with stem cells.


“Having different options to make iPSCs will be useful when scientists encounter challenges or difficulties with one approach,” said Ding, who is also a professor of pharmaceutical chemistry at the UC San Francisco. “Our approach could lead to a simpler method of creating iPSCs or could be used to directly reprogram skin cells into other cell types, such as heart cells or brain cells.”




For comparison, four transcription factors are typically used to create iPSCs using the original method. What’s more, one transcription factor typically targets thousands of genomic locations in the cell and changes gene expression at each location.

“The fact that modulating one site is sufficient is very surprising,” said Ding. “Now, we want to understand how this whole process spreads from a single location to the entire genome.”
