Publications by authors named "Oliver Muehlemann"
Article Synopsis
- Spinal muscular atrophy (SMA) is a severe childhood disease caused by mutations in the SMN1 gene, and current treatments are ineffective; researchers are exploring antisense oligonucleotides (ASOs) to enhance SMN2 function instead.
- The study focused on a specific 25-nucleotide morpholino oligomer (MO) that targets the ISS-N1 region of the SMN2 gene, testing various injection methods and dosages in an SMA mouse model.
- Results showed that this MO sequence significantly improved survival and reduced disease symptoms compared to previous options, suggesting that MO-based therapies could be a promising approach for treating SMA.