Sherman Ku- PhD: Working with Friedreich’s Ataxia and induced pluripotent stem cells (iPSC)
What is the focus of your research?
My research has focused on developing an induced pluripotent stem cell (iPSC) model of Friedreich’s ataxia (FRDA), which is an inherited neurodegenerative trinucleotide repeat expansion disorder, discovering the iPS cells themselves could be used to model repeat instability seen in brain and heart tissue in patient families. Triplet repeats in many trinucleotide repeat expansion disorders are unstable over generations, and we see it replicated with passaging of the iPS cells in this case with FRDA.
We worked to make iPS cells and differentiate them into neurons to model Friedreich’s ataxia, which hadn’t been done before.
These neurons can be used for mechanistic or drug development studies according to our proof of principle evidence, which hopefully will soon be published.
Has the iPSC model replicated the state of the disease? Is there another application for it?
Yes, we have evidence that suggests that we can replicate certain aspects of FRDA in both iPSCs and iPSC-derived neurons. Of our particular FRDA iPSC line, there is no other major application (aside from disease modelling or mechanistic studies), but certainly other groups are working on general iPSC applications such as cell or tissue replacement.
How did you first become interested in Biology?
A high school biology teacher (back in 9th grade, I think) really got me interested. First with his oddly sarcastic enthusiasm for teaching and the anecdotal stories he would tell. I remember what really piqued my interest was him telling us about early studies back in the 70s of people developing oil-metabolizing bacteria that might have been useful for oil spills… the possibility of such an application really appealed to me.
I started doing research as an undergrad in a yeast lab for three years. I really enjoyed benchwork and the objective and analytical nature of the work, so the natural step for me was to go to grad school and see where it would take me.
How does it feel to have both successfully defended AND published so recently?
It feels great to have defended and to have all that pressure off me, but at the same time, no significant changes have really occurred for me yet. Probably it’ll hit me when I leave the lab and transition to a job, but so far, things are going as they were before…just without that near-panic feeling of “I need to finish soon.” I was also really happy about having a good paper come out last fall, and I’m looking forward to hopefully another one in the next few months.


