Explored avenues for managing Spinal Muscular Atrophy (SMA) via novel therapies
In a significant breakthrough, researchers from the German Centre for Neurodegenerative Diseases (DZNE) and Dresden University of Technology are working on new treatment methods for Spinal Muscular Atrophy (SMA). This rare neurological disease, characterised by paralysis and muscle wasting, affects approximately 1,500 people in Germany who typically develop the condition in childhood.
The team's focus is on the embryonic stage of development to understand the developmental abnormalities in SMA. SMA is caused by a genetic defect that results in decreased protein production, specifically the "survival of motor neuron proteins" that nerve cells need to carry out their function of controlling movements.
The researchers have made a promising discovery: disrupted gene regulation during the embryonic stage could be a potential trigger for SMA. To further their research, they have grown organoids in the lab. These organoids mimic various stages of SMA development, including the earliest phase corresponding to a human embryo a few weeks old.
These organoids are potential resources for future research projects. If successful, the new treatment methods could be applied during early pregnancy, which could allow for earlier intervention than current treatments for SMA. It's important to note that while a gene therapy for SMA has been available for a few years, it does not provide a complete cure.
The new findings bring hope for those affected by SMA. The research team is currently conducting further research to verify the role of disrupted gene regulation in SMA and explore new therapeutic approaches based on these abnormalities. This disrupted gene regulation could be a focus for new treatment methods, offering a beacon of hope for those living with SMA.
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