Expanded therapies für SMA
In a significant breakthrough, researchers from the Dresden University of Technology and the German Center for Neurodegenerative Diseases (DZNE) are working tirelessly to understand and develop new treatment methods for Spinal Muscular Atrophy (SMA).
The team's ongoing work continues to study various stages of SMA development using organoids - rice grain-sized tissue samples with all the essential characteristics of spinal cord and muscle tissue - in the lab. These organoids mimic the earliest stages of SMA development, corresponding to a human embryo just a few weeks old.
The central finding of the tissue examinations reveals disrupted gene regulation in the embryonic stage may trigger the neurological disease SMA. The researchers are focusing on this critical stage, where developmental abnormalities are suspected to occur.
SMA is a rare neurological disease characterized by paralysis and muscle wasting. It is caused by a genetic defect that leads to a decrease in protein production, specifically the "survival of motor neuron proteins" necessary for nerve cells in the spinal cord to function properly and control movements.
A gene therapy for SMA has been available for a few years, but it does not provide a complete cure. The new research aims to address disrupted gene regulation during the embryonic stage of SMA development, potentially offering new hope for those affected by SMA, especially the approximately 1,500 people in Germany who usually develop SMA in childhood.
The potential impact of new therapeutic approaches could come into play much earlier than before, based on these findings. Application of these new methods may be during early pregnancy, according to the research.
However, it is crucial to note that further studies are necessary to confirm the findings and develop effective treatment strategies. The ongoing nature of the research ensures that progress continues to be made in the fight against SMA.
For those affected by SMA, this new research offers a glimmer of hope, bringing us one step closer to a complete cure for this debilitating disease.
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