Tuesday, 12 February 2013

Induced pluripotent stem cells shed light into ALS

In a recent study, researchers from the University of Edinburgh used skin cells from an Amyotrophic Lateral Sclerosis (ALS) patient to study the condition. Their findings give new insight on the disease, and may help scientists to better understand the already published literature, potentially leading to the development of new ALS treatments.

The researchers first took skin cells from the aforementioned patient and used them to create induced pluripotent stem cells (iPSCs). Then, they induced the iPSCs into becoming two types of cells, motor neurons and astrocytes.

Motor neurons are cells of the central neuron system (CNS) responsible for muscle activity, whereas astrocytes are star-shaped cells found in the brain and spinal cord, playing a crucial role in many functions of the CNS (e.g. to repair trauma-induced damage)

Picture of an astrocyte
An astrocyte derived from a rat

The scientists studied in vitro these cells and focused on mutations of the TARDBP gene, which produces the TDP-43 protein. Although TARDBP mutations are a rare cause of ALS, the researchers were very interested in studying the gene, as in the vast majority of ALS cases the TDP-43 protein forms pathological clumps inside motor neurons.

The researchers  found, for the first time, that the TDP protein causes the astrocytes of ALS patients to die. They also found that the motor neurons were not affected by the damaged astrocytes. This lack of motor neuron toxicity differs greatly when compared to ALS models that use a different gene associated with ALS, called SOD1. In SOD1 models, motor neurons die even when the mutated gene is present in the astrocytes alone.  

The study fails to explain what leads to motor neuron death in ALS cases with the mutated TDP43 gene. Dr. Bruijn, one of the study's chief authors, says that more research is needed to better understand the findings. According to him, the findings currently indicate that astrocyte death may actually be a key component of ALS. 

"If that is true, it should open up new therapeutic targets to support these cells so they can continue to support motor neurons." he says.

Professor Siddharthan Chandran, of the University of Edinburgh, said: 

"Motor neuron disease is a devastating and ultimately fatal condition, for which there is no cure or effective treatment. It is not just a question of looking solely at motor neurons, but also the cells that surround them, to understand why motor neurons die. Our aim is to find ways to slow down progression of this devastating disease and ultimately develop a cure."

Recent, related posts

Serio, A., Bilican, B., Barmada, S., Ando, D., Zhao, C., Siller, R., Burr, K., Haghi, G., Story, D., Nishimura, A., Carrasco, M., Phatnani, H., Shum, C., Wilmut, I., Maniatis, T., Shaw, C., Finkbeiner, S., & Chandran, S. (2013). Astrocyte pathology and the absence of non-cell autonomy in an induced pluripotent stem cell model of TDP-43 proteinopathy Proceedings of the National Academy of Sciences DOI: 10.1073/pnas.1300398110

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