Nusinersen: antisense oligonucleotide to increase SMN protein production in spinal muscular atrophy. [electronic resource]
Producer: 20171103Description: 327-337 p. digitalISSN:- 1699-3993
- Adult
- Alternative Splicing -- drug effects
- Animals
- Central Nervous System -- metabolism
- Clinical Trials as Topic
- Drug Evaluation, Preclinical
- Exons
- Gene Dosage
- Haplorhini
- Humans
- Infant
- Injections, Spinal
- Kidney Diseases -- chemically induced
- Mice
- Multicenter Studies as Topic
- Oligonucleotides -- administration & dosage
- Oligonucleotides, Antisense -- administration & dosage
- Protein Stability
- Spinal Muscular Atrophies of Childhood -- genetics
- Survival of Motor Neuron 1 Protein -- genetics
- Survival of Motor Neuron 2 Protein -- biosynthesis
- Thionucleotides -- administration & dosage
- Thrombocytopenia -- chemically induced
- Up-Regulation -- drug effects
No physical items for this record
Publication Type: Journal Article; Review
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