How a single protein helps keep our cells on track and figure out what they want to be

Helping keep our cells on track

One of the mysteries of stem cells that scientists continue to probe is how and why a stem cell becomes a more specialized form of cell. Understanding that process is key to being able to control those changes, and to being able to create the kinds of cells needed to treat different diseases and disorders.

Now CIRM-funded researchers at the University of California San Diego have uncovered a critical piece of that puzzle and it involves a protein that we have known about for quite some time.

Miles Wilkinson and Chih-Hong Lou from the UC San Diego Institute for Genomic Medicine found that a protein called UPF1 acts like a train switch in determining if a cell heads in one direction and remains in a stem cell state, or takes another track and progresses to become a neuron or brain nerve cell. Their findings are published in the journal Cell Reports.

Here comes the science lesson. UPF1 is known to be essential in the workings of the wonderfully named nonsense-mediated RNA decay pathway or NMD. NMD helps maintain the genetic health of cells, and it uses UPF1 to trigger a series of reactions in the cell that will either spur the cell to change into a neuron or stay in its present state.

In a news release about their findings Wilkinson says this could ultimately help lead to new therapies for some brain disorders such as autism and schizophrenia:

“Another implication follows from the finding that NMD is vital to the normal development of the brain in diverse species, including humans. Indeed, humans with deficiencies in NMD have intellectual disability and often also have schizophrenia and autism. Therapies to enhance NMD in affected individuals could be useful in restoring the correct balance of stem cells and differentiated neurons and thereby restore normal brain function.”

We already fund a number of research projects focused on autism so these findings might help speed that research along


kevin mccormack