Keeping a clear mind
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Exciting developments
“Very exciting” is how Dr. Ackerman describes a recent research project that attracted widespread interest from the neurobiology community and the science media when it was published in the September 7, 2006, issue of Nature. The focus is protein misfolding, familiar to many as the signature process in Alzheimer’s disease that clogs brain neurons with a toxic protein sludge and triggers cell death.
In her experiments, Dr. Ackerman used another mouse— known as “sticky”—that displays movement problems as an adult due to neuron loss in the cerebellum. And, surprisingly, she found that the neurodegeneration was caused not by a mutated gene, but by a mechanism involving errors in protein synthesis in brain cells. This mild “editing” defect, described for the first time in a mammalian system, results in malformed proteins and symptoms similar to those associated with neurodegenerative disease in aging humans.“We think that fully understanding this process could lead to treatment, to stopping or slowing the progression of neurodegenerative disease in people caused by protein misfolding,” Dr. Ackerman says. Her laboratory is now pursuing so-called modifier genes that can override the editing defect, and recently identified a modifier that suppresses neurodegeneration in sticky mice and could help identify a similar protective mechanism in humans.
The Nature publication came shortly after her HHMI appointment. In 2005 Dr. Ackerman joined colleague Simon John as one of the Laboratory’s two HHMI investigators and the only such honorees in Maine. Designed to spur scientific discovery by the nation’s most talented and innovative scientists, the program provides unrestricted research funding for five years, freeing recipients from the timeconsuming burden of seeking other grants.
“It’s an incredible vote of recognition for our science,” Dr. Ackerman says. “It has been wonderful to have the additional funds to use on new projects in the lab, and to have been affiliated with, and to meet, some of the most amazing scientists in the world.”
