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ISSCR honors UCSF cancer scientist with 2011 Outstanding Young Investigator Award

April 21, 2017

Using microRNAs for therapeutic purposes has great potential , he said. "They could be used either to induce adult cells to de-differentiate to embryonic stem cells, which could be expanded, manipulated and returned to a patient, or to promote differentiation of embryonic stem cells to produce tissues that would remain integrated in the body once re-introduced." They also could be used to target cancers, and they attract interest from biotechnology companies.

Because microRNAs are small molecules, he noted, they could be delivered in ways similar to everyday drugs, but would likely have far more direct effects than artificially created small molecules, "as millions and millions of years of evolution have molded them to have specific effects on the cells."

Moreover, because they are transient, he said, they could be introduced, have an impact and then be gone in a couple of days. Like a drug, he said, they would be metabolized and not create a permanent genetic change.

"Robert has made significant insights into the molecular regulation of stem cell self-renewal, differentiation, and dedifferentiation, and is helping lay the groundwork for potential therapeutic strategies," said Arnold Kriegstein, MD, PhD, director of the Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research at UCSF.

His microRNA research is part of his overall interest in the molecular tools that "lock in" differentiation of adult cells. Tight regulation is important for maintaining organ function and avoiding the possibility of uncontrolled cell growth, the basis of cancer. But learning how to unlock the differentiated state would offer potential paths to regenerative medicine and cancer therapies.

"Along with research on DNA methylation and histone modifications, insights into microRNAs should lead to a deeper understanding of the role of epigenetics in development and disease," he said. "We expect they'll all be tied together."

Source: University of California - San Francisco