Awakening a heart ribosome in the brain: preventing and treating neurodegenerative diseases with ribosome reprogramming

Our vision is to uncover the fundamental principles of how genetic information is coded in the genome and decoded in cells in both health and disease, and to develop methods to control these processes in cancer, heart disease, aging, and neurodegeneration. To achieve these goals, we harness cutting-edge technologies such as CRISPR, genomics, and machine learning.

Xuebing Wu, PhD is an Assistant Professor in the Department of Medicine and the Department of Systems Biology at Columbia University Irving Medical Center. Dr. Wu received a B.S. and M.S. in Control Science and Engineering from Tsinghua University, Beijing and a PhD in Computational and Systems Biology from MIT, working with Phillip Sharp and Christopher Burge. As a Helen Hay Whitney Fellow, he continued postdoctoral work on functional genomics of RNA structures with David Bartel at Whitehead Institute/MIT. Research in the Wu laboratory integrates CRISPR, genomics, and machine learning to decode and target the central dogma in human health and disease. Dr. Wu has received a number of other distinctions including The RNA Society/Scaringe Young Scientist Award, Pew-Stewart Scholars Award, NIH Director’s New Innovator Award, and Pershing Square Sohn Prize.

Most neurodegenerative diseases are caused by the buildup of misfolded proteins in the brain as we age. While current treatments primarily focus on clearing these toxic proteins, me and my team are taking a different approach. We aim to reduce the production of these toxic proteins by reprogramming the ribosomes in the brain. This innovative strategy has the potential to not only treat but also prevent diseases like Alzheimer's and other neurodegenerative disorders.

"The MIND Prize will enable us to pursue a bold idea with the potential to revolutionize the treatment of Alzheimer's and other neurodegenerative diseases. This high-risk project is something that few funding agencies are willing to support, making the MIND Prize crucial for its advancement."