Skin cells turned into working brain cells could mean new hope for Alzheimer's patients
A breakthrough in cell engineering could mean good news for drug developers targeting Alzheimer's, Parkinson's and other brain diseases.
With the introduction of just one gene, scientists at San Francisco's Gladstone Institute successfully turned skin cells into fully functioning brain cells.
In their study, published in Cell Stem Cell, researchers describe how they introduced a single gene called Sox2 into both mouse and human skin cells. Within days, the skin cells transformed into early-stage brain cells, which then began to self-renew. They soon matured into neurons capable of transmitting electrical signals and within one month had formed neural networks.
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This Frankenstein-like feat could be useful for testing out potential Alzheimer's treatments. There are still no approved drugs to treat the disease.
"Many drug candidates-especially those developed for neurodegenerative diseases-fail in clinical trials because current models don't accurately predict the drug's effects on the human brain," said Dr. Huang, who heads the laboratory at Gladstone where the work was completed.
By testing drugs in these artificially grown brain cells, Huang said he hopes to speed up the drug discovery process for these debilitating diseases.
The idea, according to Yadong Huang, head of the Gladstone study, is to scrape cells from an Alzheimer's patient's skin, turn it into a brain cell, and try out different therapies on the newly-created cells. If there's an adverse reaction, doctors will know not to give that drug to the patient, reports U.S. News & World Report.
"Human neurons-derived from reengineered skin cells-could help assess the efficacy and safety of these drugs, thereby reducing risks and resources associated with human trials," he said.
The researchers took special care to engineer these cells with specific genetic factors that wouldn't allow for 'rogue' cell renewal that could lead to tumors.
"We wanted to see whether these newly generated neurons could result in tumor growth after transplanting them into mouse brains," said lead author Karen Ring of University of California, San Francisco. "Instead we saw the reprogrammed cells integrate into the mouse's brain-and not a single tumor developed."
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