Welcome to our Summer 2015 newsletter.  For more updates, join us on  Facebook Twitter or our  SCRMC website.

SCRMC Faculty Make Summer News

Clockwise from top left: President Barack Obama has named Judith Kimble, Ph.D., professor of biochemistry, to chair the President's Committee on the National Medal of Science; Emery Bresnick, Ph.D., Kellett professor of cell and regenerative biology, has identified dozens of genes related to blood cell development and function; Brainxell is a new startup exploring treatments for neurological disorders by Su-Chun Zhang, Ph.D., Steenbock professor of neuroscience; the American Veterinary Association has given its 2015 Lifetime Achievement Award to Ian Duncan, Ph.D., B.V.M.S., professor of neurology. Read more UW-Madison news about SCMRC members here.

Summer Science Camp is Nationally Lauded

Morgridge Institute's Summer Science Camp received national news media attention. The camp has proven to be a showcase of UW-Madison's leadership and top talent in stem cell science, including contributions from the Stem Cell and Regenerative Medicine Center, Wisconsin Stem Cell Roundtable, School of Medicine and Public Health, College of Engineering and others. ( Photo by Courtni Kopietz )

Researchers Tackle Fragile X Disorder

By Jordana Lenon

Fragile X syndrome (FXS) is the most common inherited intellec-tual disability and greatest single genetic contributor to autism.

Through two revealing studies at the Waisman Center, SCRMC member Xinyu Zhao, Ph.D. (right) and SCRMC Neural Regeneration Focus Group Chair Anita Bhattacharyya, Ph.D. ( left), pictured with their post-doc Meng Li, Ph.D. ( center), are advancing our understanding of this disorder and exploring new treatments.

FXS affects one in 4,000 males and one in 6,000 females. It is linked to an X chromosome gene mutation and shutdown that disrupts normal production of a critical protein, FMRP. 

Children with FXS often have deficits in working memory and low IQs, are more prone to anxiety, attention deficit disorder and autism, and display physical features such as a prominent jaw and forehead, and a long, narrow face.

Left: Mouse models of FXS have impaired performance in learning and memory tasks. Their neurons do not mature properly and are not well connected to other neurons. Image A is a normal neuron; B is abnormal. (Doers et al, 2014)

In the first study, involving mouse genetics and published June 4 in Cell Reports , Zhao, SMPH  professor  of neuroscience, showed that two proteins might actually be involved. FMRP and another suspect protein, FXR2P, while acting through separate mechanisms in new neurons, may also work together to promote neural development. 

The findings suggest that manipulating one or both proteins through drugs that foster new nerve cell development postnatally may help those with fragile X syndrome and other disorders involving malformed neurons.

A second Waisman Center FXS study involves screening drugs on neurons grown from pluripotent stem cells. This project involves three phases of research and has just been awarded additional funding from the John Merck Fund.

First, Bhattacharyya, Waisman Center senior scientist, with help from SCRMC member Su-Chun Zhang, Ph.D., derived human iPS cells and their neural derivatives from individuals with FXS. Second, with a grant from the FRAXA Research Foundation, she and Zhao attempted to test known drugs that could reactivate the fragile X genes in these cells, but without success. In phase three, with a pilot grant from the John Merck Fund and assistance from SCRMC member Kris Saha, Ph.D., she and Zhao, together with their post-doc Meng Li, Ph.D., created highly sensitive "reporter cells" through gene editing, so that the FXS cells can "report" when their shut-down gene gets reactivated.

Now, with additional Merck funding, Zhao and Bhattacharyya are screening thousands of small molecule-based drugs using their reporter cells, to try and identify a drug that might cause gene reactivation. The next step will be preclinical testing in mice, with hopes of developing a breakthrough clinical therapy.


Fast Fact

Wisconsin is home to more than 40 companies engaging in stem cell and regenerative medicine activities and employing over 3,500 people.


Did You Know?

This month marks the 20-year anniversary of the first successful derivation and culture of a primate embryonic stem cell line. Read the original PNAS paper by Thomson et al.


Five are Summer Undergraduate Fellows

Congratulations to the following for completing the  Summer Undergraduate Research Fellowship (SURF)  program with their graduate student mentors this summer: Alex Waldman, mentored by Qian Bu in Qiang Chang's medical genetics and neurology lab at the Waisman Center; Drew Richards, mentored by Matt Stebbins in Sean Palecek's and Erik Shusta's chemical and biological engineering and biomedical engineering labs; Meng Lou, mentored by Jared Carlson-Stevermer in Kris Saha's biomedical engineering lab at the Wisconsin Institute for Discovery (WID); Jaime Brown, mentored by Nur Zafirah Zaidan in Rupa Sridharan's cell and regenerative biology lab at WID; and Wanying Lou, mentored by Matt Brown in Will Burlingham's surgery lab in the School of Medicine and Public Health. The SURF program is run by the Wisconsin Stem Cell Roundtable (WiSCR) with SCRMC support.

Save the Date!


SCRMC Fall Conference

Friday, Sept. 25, 2015

Discovery Building: 

Scheduled keynote speakers are Amy Wagers, Ph.D., Harvard University Department of Stem Cell and Regenerative Biology, Joslin Diabetes Center and Harvard Medical School; 

and Karen Downs, Ph.D., 

SCRMC member and UW-

Madison Department of Cell

and Regenerative Biology.