Study highlights the potential of cell replacement therapy for Huntington's disease

New research appearing in the journal Nature Biotechnology answers important questions about the viability of treatments that seek to replace diseased and aged cells in the central nervous system with healthy ones. Its findings have implications for a number of neurological and psychiatric disorders-;including Huntington's disease, amyotrophic lateral sclerosis (ALS), and schizophrenia-;that have been linked to glia, a population of cells that support brain health and function.

The new study from the URMC Center for Translational Neuromedicine, which Goldman co-rects, describes the ability of human glial progenitor cells–precursor cells that can give rise to both astrocytes and oligodendrocytes, the two major types of glia-;to compete with one another in the adult brain, and the competitive advantage of young and healthy cells over aged and diseased cells.

The culmination of a decade of scientific progress

A number of recent important advances are behind the new findings. In 2013, Goldman and colleagues first reported strategies for producing the brain's glial support cells from embryonic stem cells. In later research, the lab transplanted these cells into the brains of baby mice, resulting in the creation of human glial-chimeric mice, a technical achievement that enables the researchers to study human glial cells in the living brain. The team showed that after transplantation, the human glial progenitor cells quickly outcompeted native cells, resulting in brains with mouse neurons and human glia

In later experiments, the lab transplanted human glial cells with the Huntington's disease (HTT) mutation. They observed that this mutation impaired the function of glial progenitor cells, resulting in poor astrocytes and oligodendrocytes production. The lab also showed that transplanting healthy human glial progenitor cells into mouse models of Huntington's delayed disease progression, reinforcing the importance role that glial dysfunction plays in this still untreatable neurodegenerative disease.

Young and healthy cells outcompete older and sick ones

As these prior studies were limited to the transplant of human cells into the mouse brain, the question remained whether human cells transplanted into another human brain would yield the same type of benefit. The new Nature Biotechnology study strongly suggests that the answer to this question is yes, and highlights the potential value of cell replacement therapies by showing that healthy human glia will outcompete and replace sick human cells.

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