Spinal muscular atrophy (SMA) is a severe neurological disease without a cure, affecting about 1,500 individuals in Germany.
Scientists in Dresden are studying organoids to uncover abnormalities in embryonic development linked to SMA.
Organoids grown from stem cells mimic spinal cord and muscle tissue, revealing premature neuron development in SMA.
Correcting the genetic defect in SMA only partially improves developmental abnormalities, suggesting additional treatment is needed.
Targeting gene regulation through epigenetics may lead to improved therapies for SMA, potentially applied in early pregnancy.
Researchers in Dresden, Germany are shedding new light on spinal muscular atrophy (SMA), a severe neurological disease that affects around 1,500 individuals in Germany. While current therapies can alleviate symptoms, there is no cure. By studying organoids – miniaturized tissue cultures grown in the lab – scientists have uncovered previously unnoticed abnormalities in the embryonic development of SMA. Specifically, they found that stem cells in SMA organoids tended to develop prematurely into spinal cord neurons, leading to developmental defects. These findings, published in Cell Reports Medicine, suggest that treatments targeting gene regulation may be needed to address SMA more effectively.
The researchers’ work with organoids, which mimic the spinal cord and muscle tissue, revealed key insights into the underlying mechanisms of SMA. By observing abnormal cellular development in the organoids, the scientists believe that targeting gene regulation may be a promising avenue for future treatments. Correcting the genetic defect associated with SMA only partially improved the developmental abnormalities, indicating that a more comprehensive approach is needed. Looking ahead, the researchers suggest that therapies involving epigenetic drugs targeting gene regulation may be beneficial in addressing the early neurodevelopmental defects seen in SMA. These findings offer hope for better treatment options for individuals affected by this debilitating disease.
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