A half-century ago, James Watson and Francis Crick became renowned for revealing the double helix structure of DNA, the molecule in each cell that provides the “blueprint” for human beings. This breakthrough laid the foundation for our understanding of how information is transferred in biology.
Arguably one of the most profound scientific discoveries of the 20th century — solving the DNA structure — required the collaboration of scientists from the physical and biological sciences.
Now, a collaboration at the University of Michigan has set out to bring insights from the molecular realm of RNA to bedside medicine. Rapidly emerging insights into how RNA, DNA’s close chemical cousin, helps modify, safeguard and translate genetic information in each of us is offering the promise of a personalized “precision medicine.”
Most current medical treatments are one-size-fits-all, an approach that is successful for some patients but not others. Precision medicine is a modern approach to disease prevention and treatment that takes into account individuality, which is shaped by genetic inheritance, environment and lifestyle. While DNA provides the genetic code for a human life, it is RNA that determines which parts of the code are used and when. Over the lifespan of a human being, the parts of the DNA blueprint that are read out by RNA constantly change, leading to aging and disease.
Current discoveries in RNA biomedicine are paving the way for diagnostics that can detect problems early and therapies that fix problems at their roots, rather than simply treating symptoms. The premise of RNA-focused precision medicine is that human diseases, such as cancer and Alzheimer’s, could be cured with less invasive, more targeted treatments with minimized side effects, tailored to each of us based on our RNA makeup.
In genetic diseases, segments of the human genome are reorganized in ways that can derail normal function. A notable example of RNA in precision medicine is the recently FDA-approved treatment for spinal muscular atrophy, a neuromuscular disease that is the leading genetic cause of death in infants. A research group led by Prof. Adrian Krainer at the Cold Spring Harbor Laboratory in New York — incidentally, where Watson went on to serve as director and president — collaborated to study RNA splicing defects, or errors in editing the RNA sequences. The team was able to successfully correct a splicing defect that caused SMA by using the revolutionary therapeutic approach of injection of a stretch of modified RNA that corrected the defect, allowing the cells to make a protein necessary to keeping muscle nerve cells functioning. This groundbreaking therapy can save lives.
In the spirit of collaboration across the physical and biological sciences, University President Mark Schlissel has urged University researchers to embrace the potential of precision medicine, as the University’s faculty are leaders in this emerging field. One group addressing the molecular aspects of precision medicine is the members of the University’s Center for RNA Biomedicine. The CRB started in spring 2016, promoting and developing interdisciplinary collaborations across the University by bringing researchers together for bi-weekly seminars showcasing RNA studies from the physical to the clinical sciences, as well as for an annual symposium hosting RNA experts from around the world. This year, the Center is also funding four pilot grants, totaling $300,000, to forge new teams among UM researchers.
On March 31, 2017, the CRB will host its second annual symposium, “RNA in Precision Medicine,” which will feature speakers in the fields of RNA diagnostics and therapeutics. All seminars and symposia are free and open to the University community. For more details on the CRB and how to join these collaborative efforts, visit the CRB’s website. Who knows? You may be the next Watson or Crick and will make a lasting impact in precision medicine by discovering and targeting the RNA underlying human disease.
On behalf of the Center for RNA Biomedicine,
Nils G. Walter, Founding Co-Director
Martina Jerant, Administrative Coordinator
Suzanne Tainter, Web and Publications Specialist, Chemistry Department