New Approaches to Treating ALS

(Stem Cells News image)

Researchers at University of Exeter Medical School in the United Kingdom have started an ambitious project to investigate how mitochondria flaws may cause the neuron cell death associated with ALS.

Not only may their insights provide a better understanding of how amyotrophic lateral sclerosis arises, but it also may lay a foundation for the development of new treatments.

Mitochondria are the cell’s power plants, converting nutrients to energy. Researchers believe abnormalities in their functioning may contribute to ALS.

The team, led by Dr. Akshay Bhinge of Exeter’s Living Systems Institute, noted that mutations in the TBK1 and OPTN genes were recently linked to ALS. The genes take part in a cell process called mitophagy, or the removal of defective mitochondria.

Although studies suggest that mutations in the genes can cause disease, researchers have no idea how.

To find out, the research team plans to use induced pluripotent stem cells. These are stem cells that, in the lab, are forced back into development to become stem cells. Researchers will use a gene editing tool called CRISPR-Cas9 to introduce mutations they find in patients (…)


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