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Science AMA Series: I am Dr. Nikolay Dokholyan, professor at the University of North Carolina School of Medicine in Chapel Hill, N.C., here to talk about a major development toward understanding ALS an
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Hi Reddit, As the Michael Hooker Distinguished Professor of Biochemistry and Biophysics at the UNC School of Medicine, I study the causes of human diseases such as cystic fibrosis and amyotrophic lateral sclerosis (ALS). Every day, an average of 15 people are newly diagnosed with ALS. That’s more than 5,600 people per year. Annually, ALS, also known as Lou Gehrig’s disease, is responsible for two deaths per 100,000 people. Along with my colleagues, I recently completed some research on ALS that could lead to significant developments in how we treat the disease. In my lab, we approach research very differently than many other labs. We use integrated strategies to replicate molecular structural modeling. This way, when we analyze the structure and dynamics of biological molecules, they are at consistent time scales to actual biological systems. This is also how we approached our ALS research. Although there has been a significant amount of research on ALS, the exact form of the aggregated protein responsible for killing neurons has been hard to identify – and even harder to study. To crack the mystery, our team used a combination of computational modeling and experiments in live cells. We spent two years developing a custom algorithm to determine the molecules’ structure, which is an outstanding feat. Next, we spent several more years developing methods to test the trimers’ effect on motor neuron-like cells. The results of our study, published in Proceedings of the National Academy of Sciences, show the first definitive evidence that these protein clumps are indeed toxic to the type of neurons that die in patients with ALS. Our findings raise a lot of questions about what this could mean for halting the progression of the disease and, eventually, developing its treatment. I will be back at 1 pm EST (10 am PST, 6 pm UTC) to answer your questions, Ask me anything! Edit: Thank you for all the great questions! I’m signing off!