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Antisense Oligonucleotide Development for Treatment of Spinal Muscular Atrophy
TS-037741 — The SMN2 gene contains sequences that regulate the level of inclusion of SMN exon7 in SMN mRNA, and some of these act in a negative manner. Antisense oligonucleotides to block the negative regulating protein binding to these sites can increase inclusion of SMN exon 7 and thus, the amount of SMN being produced.
Spinal muscular atrophy (SMA) is a genetic disorder that affects the control of muscle movement. It is caused by a loss of motor neurons in the spinal cord and brainstem, leading to weakness and atrophy in the muscles used for walking, crawling, sitting up, and head movement. In severe instances, …
  • College: College of Medicine (COM)
  • Inventors: Burghes, Arthur; McGovern, Vicki; Prior, Thomas
  • Licensing Officer: Flammang, Ann Marie

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