Antisense Oligonucleotide-Mediated Terminal Intron Retention of the SMN2 Transcript

• Main Authors: Loren L. Flynn, Chalermchai Mitrpant, Ianthe L. Pitout, Sue Fletcher, Steve D. Wilton
• Other Authors: Murdoch University
• Format: Article
• Published: 2019
• Subjects: Biochemistry, Genetics and Molecular Biology; Pharmacology, Toxicology and Pharmaceutics
• Online Access: https://repository.li.mahidol.ac.th/handle/123456789/45150
• Institution: Mahidol University

© 2018 The Authors 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 criti...