A stem cell, an example of disease
For the first time they have turned a stem cell into an example to investigate a genetic disease. A sick patient is removed from a superficial cell and reprogrammed for use in research. The skin cell has become motor neuron in the laboratory and the newly created neuron has acted as the neurons found in the patient's brain, expressed by the same gene.
The disease is a spinal muscular atrophy, a type of degeneration of neurons that manage movement. It paralyzes the patient and can even cause death in very serious cases. This is a very rapid disease, so it is difficult for laboratory animals to serve as an example for their research. The solution has been to model a cell instead of an animal.
Scientists will use this cell to test medications. In the case of this muscular atrophy, it is about testing two molecules, balproic acid and tobramycin.
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Zientzia aldizkaria
