At UW-Madison, 500 scientists studying stem cells 20 years after discovery | Local News

Ashton's three-dimensional neural tubes in a dish could also be used to screen chemicals and environmental toxins to see if they kill brain cells or alter cells in ways that cause birth defects.

"Ashton, an assistant professor of biomedical engineering at UW-Madison, said:" It could be a useful model for the development process.

At the university, Ashton is one of 100 faculty researchers – and among about 500 scientists overall – who are using stem cells for drug or chemical screening, or who is working with Dr. Tim Kamp, director of the university's Stem Cell and Regenerative Medicine Center.

James Thomson first named the human embryonic stem cells, which campus scientist. In vivo stem cells – skin or blood cells reprogrammed to their embryonic state – which Thomson and Japanese researcher Shinya Yamanaka developed in 2007.

Ashton was drawn to stem cells because of Thomson's pioneering role in the field. In 2001, when Ashton was in college studying chemical engineering, Time magazine featured Thomson on the cover.

Ashton reads the article and decides to devote his career to applying engineering principles to stem cells. "I thought, 'How do we make them into useful products?'"

Krishanu Saha, also an assistant professor of biomedical engineering, uses CRISPR, a form of gene editing.

CRISPR, which stands for clustered interspaced short palindromic repeats, is a "molecular scissor" that allows genes to be precisely edited, potentially as a therapy. Discovered in 2012, the technique is based on a defense mechanism in bacteria.

Saha has packaged CRISPR editing machinery into a nanoparticle that could be injected into patients to repair disease genes. He is working with Dr. David Gamm to develop gene editing for macular degeneration, among other potential clinical uses.

Gene editing can cause unintended changes in the genome, which could lead to immune reactions or even cancer. Saha uses stem cells, taking advantage of their ability to grow indefinitely in a dish.

"You can systematically test the genome with the same cell lines," he said.

Another condition Saha's strategy could benefit is Fragile X syndrome, a developmental disorder that is a focus of Anita Bhattacharyya's research.

Bhattacharyya, an assistant professor of biology at UW-Madison's Waisman Center, is studying iPS cells from Fragile X patients and embryonic stem cells, which have been modified by CRISPR, with Saha's help, to carry the gene mutation that causes the condition. In people with the disorder, the gene is turned off.

"We're looking for ways to turn the gene back on, which might relieve some of the symptoms," Bhattacharyya said.

She is also studying iPS cells of people with Down syndrome, to understand why they have a shortage of nerve cells and tend to develop Alzheimer's disease. The hope is not for a cure, but for a treatment that might help patients live more independently.

"We can use our iPS-cell-derived neurons to ask, if we can correct oxidative stress, does that lead to better function?" She said.