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Amyotrophic Lateral Sclerosis

Now, human motor neurons from stem cells

The degeneration of neurons responsible for motor activity, known as motor neurons, leads to paralysis and even death in many diseases. However, scientists may have found a way to help the body manufacture replacement neurons for these dying cells according to a new research report.

The breakthrough has been published in Nature Biotechnology by researchers at the Institute for Stem Cell Therapy and Exploration of Monogenic Diseases who in collaboration with CNRS and Paris Descartes University succeeded in producing different populations of motor neurons in only 14 days by using a different approach to controlling the differentiation of human pluripotent stem cells. Pluripotent stem cells are master cells that can make any cell or tissue the body needs to repair itself.

This discovery is significant because it may enable scientists to mass produce these neurons, which, in turn would allow more rapid progress in our understanding of diseases of the motor system, such as infantile spinal amyotrophy or amyotrophic lateral sclerosis (ALS). Put simply, it promises to enhance our ability to understand and fight degenerative neural diseases and evolve superior therapeutic strategies.

Owing partly to poor understanding of the molecular mechanisms that control the differentiation of specialized cells like the motor neurons, scientists have long struggled to derive them from human pluripotent stem cells in an efficient and targeted manner.

“Generally, the targeted differentiation of human pluripotent stem cells is a long and inefficient process. Now, however, with our new approach we have succeeded in obtaining these neurons in only 14 days, nearly twice as fast as before and with a rarely achieved homogeneity,” explains Cécile Martinat, an Inserm Research Fellow at I-Stem.

The ‘new approach’ evolved from an intense study of the interactions between some molecules that control embryonic development. This enabled the researchers to better understand the mechanisms that govern the generation of these neurons during development and also find an optimal “recipe” for producing them efficiently and rapidly.

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