Nusinersen: antisense oligonucleotide to increase SMN protein production in spinal muscular atrophy.

Paton, D M

Nusinersen: antisense oligonucleotide to increase SMN protein production in spinal muscular atrophy. [electronic resource] - Drugs of today (Barcelona, Spain : 1998) Jun 2017 - 327-337 p. digital

Publication Type: Journal Article; Review

1699-3993

10.1358/dot.2017.53.6.2652413 doi


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