TY  - GEN
AU  - Paton,D M
TI  - Nusinersen: antisense oligonucleotide to increase SMN protein production in spinal muscular atrophy
SN  - 1699-3993
PY  - 2017///1103
KW  - Adult
KW  - Alternative Splicing
KW  - drug effects
KW  - Animals
KW  - Central Nervous System
KW  - metabolism
KW  - Clinical Trials as Topic
KW  - Drug Evaluation, Preclinical
KW  - Exons
KW  - Gene Dosage
KW  - Haplorhini
KW  - Humans
KW  - Infant
KW  - Injections, Spinal
KW  - Kidney Diseases
KW  - chemically induced
KW  - Mice
KW  - Multicenter Studies as Topic
KW  - Oligonucleotides
KW  - administration & dosage
KW  - Oligonucleotides, Antisense
KW  - Protein Stability
KW  - Spinal Muscular Atrophies of Childhood
KW  - genetics
KW  - Survival of Motor Neuron 1 Protein
KW  - Survival of Motor Neuron 2 Protein
KW  - biosynthesis
KW  - Thionucleotides
KW  - Thrombocytopenia
KW  - Up-Regulation
N1  - Publication Type: Journal Article; Review
UR  - https://doi.org/10.1358/dot.2017.53.6.2652413
ER  -