Antisense Oligonucleotide-Mediated Terminal Intron Retention of the SMN2 Transcript

• Main Authors: Loren L. Flynn (Author), Chalermchai Mitrpant (Author), Ianthe L. Pitout (Author), Sue Fletcher (Author), Steve D. Wilton (Author)
• Format: Book
• Published: Elsevier, 2018-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

The severe childhood disease spinal muscular atrophy (SMA) arises from the homozygous loss of the survival motor neuron 1 gene (SMN1). A homologous gene potentially encoding an identical protein, SMN2 can partially compensate for the loss of SMN1; however, the exclusion of a critical exon in the cod...