Targeting the 5′ untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy
- Resource Type
- Article
- Authors
- Winkelsas, Audrey M.; Grunseich, Christopher; Harmison, George G.; Chwalenia, Katarzyna; Rinaldi, Carlo; Hammond, Suzan M.; Johnson, Kory; Bowerman, Melissa; Arya, Sukrat; Talbot, Kevin; Wood, Matthew J.; Fischbeck, Kenneth H.
- Source
- In Molecular Therapy - Nucleic Acids 5 March 2021 23:731-742
- Subject
- Language
- ISSN
- 2162-2531