What do the lowly yeast and human stem cells have in common?
They’re ingredients in a discovery platform that might lead to a way to stop neurodegenerative diseases like Alzheimer’s, Parkinson’s and ALS, said Susan Lindquist, Ph.D., winner of the 2014 Vanderbilt Prize in Biomedical Science.
Last month Lindquist, a pioneer in protein folding science at the Whitehead Institute for Biomedical Research and professor of Biology at MIT, described her efforts to unravel the secrets of diseases precipitated by proteins that “misfold.”
“Every protein has to fold into exactly the right shape in order to function,” she explained during a Flexner Discovery Lecture.
“If they don’t get this exactly right, they won’t function and will cause horrible diseases,” she said. “But even worse, if they misfold in certain ways they can go off and do renegade things.”
Yeast has long been used as a “model organism” to study human genes and proteins. Recently scientists have learned how to induce adult human stem cells to develop into any tissue, including nerves.
Lindquist and her colleagues model misfolding pathologies in yeast and test compounds for their ability to correct the pathologies. The compounds then are tested in human neurons, produced from the stem cells of patients with disease-causing genetic mutations.
A company Lindquist cofounded last year called Yumanity Therapeutics is using platforms she developed to identify potential new targets for neurodegenerative diseases.
Lindquist is the ninth recipient of the Vanderbilt Prize, which recognizes women scientists who have an outstanding record of research and who mentor other women in science. Prize winners mentor women graduate students at Vanderbilt who are called Vanderbilt Prize Scholars.
This year’s scholar, Jessica Moore, works in the labs of Eric Skaar, Ph.D., MPH, who directs the Program in Microbial Pathogenesis, and Richard Caprioli, Ph.D., director of the Vanderbilt Mass Spectrometry Center.
Moore is applying imaging mass spectrometry to animal models of infectious disease to uncover novel host and bacterial factors contributing to the outcome of infection. In their nomination letter, Skarr and Caprioli wrote that she was a “born leader.”
“Few students have the creativity, patience and dedication to move seamlessly between two laboratories with such distinct expertise,” they wrote. “What Jessica has been able to accomplish independently usually requires an entire team of scientists.”
For more information about the Vanderbilt Prize, go to https://medschool.vande rbilt.edu/dean and click on “Vanderbilt Prize.”
For a complete schedule of the Discovery Lecture series and archived videos of the lectures, visit www.mc.vanderbilt.edu/discoveryseries.