— 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 mann
er. 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, …