Washington DC: Researchers devised a process to convert a skin cell directly into a neuron, eliminating the need to generate induced pluripotent stem cells. Such neurons could be used to treat spinal cord injuries or diseases such as Amyotrophic Lateral Sclerosis (ALS).
Converting one type of cell to another — for example, a skin cell to a neuron — can be done through a process that requires the skin cell to be induced into a “pluripotent” stem cell, and then differentiated into a neuron.
Researchers at the Massachusetts Institute of Technology (MIT) have now devised a simplified process that bypasses the stem cell stage, converting a skin cell directly into a neuron.
Working with mouse cells, the researchers developed a highly efficient conversion method that can produce more than 10 neurons from a single skin cell. If replicated in human cells, this approach could generate large quantities of motor neurons, which could potentially be used to treat patients with spinal cord injuries or diseases that impair mobility.
“We were able to get to yields where we could ask questions about whether these cells can be viable candidates for the cell replacement therapies, which we hope they could be. That’s where these types of reprogramming technologies can take us,” said Katie Galloway, the WM Keck Career Development Professor in Biomedical Engineering and Chemical Engineering.
As a first step toward developing these cells as a therapy, the researchers showed that they could generate motor neurons and engraft them into the brains of mice, where they integrated with host tissue.
Galloway is the senior author of two papers describing the new method, which appears today in Cell Systems. MIT graduate student Nathan Wang is the lead author of both papers.
The MIT team also hopes to increase the efficiency of this process for human cell conversion, which could allow for the generation of large quantities of neurons that could be used to treat spinal cord injuries or diseases that affect motor control, such as ALS.
Clinical trials using neurons derived from Induced pluripotent stem cells (iPSCs) to treat ALS are now underway, but expanding the number of cells available for such treatments could make it easier to test and develop them for more widespread use in humans, Galloway says.
The research was funded by the National Institute of General Medical Sciences and the National Science Foundation Graduate Research Fellowship Program.
