Dresden Scientists Unveil New SMA Treatment Hope
Scientists at the University of Dresden are making strides in understanding and treating spinal muscular atrophy (SMA), a genetic disorder that causes muscle wasting and paralysis. Around 1,500 children in Germany are affected by SMA each year.
The research team, affiliated with the German Center for Neurodegenerative Diseases (DZNE) and the Technical University of Dresden (TU Dresden), is exploring new treatments. They are focusing on the embryonic stage, as disrupted gene regulation at this point may trigger SMA.
In the lab, researchers have been culturing organoids to study various stages of SMA development. This work could pave the way for treatments applicable during early pregnancy. The disease is caused by a genetic defect that reduces the production of 'survival of motor neuron proteins'. Existing gene therapy provides relief but falls short of a full cure.
The team's findings could significantly improve SMA treatment. By understanding and targeting the embryonic stage, they aim to develop more effective therapies. This research brings hope to the families affected by this devastating condition.
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