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Latest Stem Cell Discovery – A New Hope For ALS Patients!

A team of scientists from the University of California, San Diego School of Medicine, with the help of iPSCs or induced pluripotent stem cells extracted from people suffering from ALS (amyotrophic lateral sclerosis) has shown that inheritance of one form of ALS is linked to decreased levels of a particular protein in their bodies.

Amyotrophic lateral sclerosis is a fatal, progressive and an incurable neurodegenerative condition affecting nearly 5000 people from the USA alone every year. It is characterized by the death of neurons or nerve cells in the brain and spinal cord responsible for the voluntary muscle movement including movement of the arms, legs and body. 10% of the people affected, inherit the condition from a family member.

Although several studies in the past have succeeded in testing drugs against ALS in rodent models, none of them were efficient enough on humans tested during pre-clinical and clinical trials.

The current study involved a group of patients suffering from a familial (inherited) form of ALS known as ALS8 from 2 independent families. Induced pluripotent stem cells from their skin cells were derived from which neurons were created. This served as a laboratory model of the disease.

Moreover, the mutated gene responsible for the disease development could be tracked during the differentiation of neurons.

According to Alysson R. Muotri, one of the senior authors of the study, ALS8 is a result of the mutations (permanent changes in the DNA) in a gene called VAPB. Through this study, it was found that the levels of VAPB protein are decreased in the motor neurons developed from the iPSCs of ALS8 patients when compared to their siblings’ cells who were not the victims of ALS8.

Although the team is yet to find a connection between the low levels of VAPB and the development of ALS, this new model will enable to develop diagnostic methods and drugs against it.

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