Atrofia Muscular Espinal/terapia; Animales; Humanos

ALS; ASO; Antisense oligonucleotide; BBB; CREB; FDA; Food and Drug Administration; GSK-3; Genetic disease therapy; HDAC; ICV; IGF-1; IP; ISS-N1; ISS-N2; IV; JAK; Janus kinase; N-methyl d-aspartic acid; NMDA; NSAID; SAHA; SC; SMA; SMA therapeutics; SMN; STAT5; Spinal muscular atrophy; Splicing; Survival motor neuron; T-cell restricted intracellular antigen 1; TIA1; TSA; VPA; amyotrophic lateral sclerosis; antisense oligonucleotide; blood brain barrier; cyclic AMP response element binding protein; glycogen synthase kinase 3; heterogenous ribonucleoprotein A1; histone deacetylase; hnRNP A1; insulin-like growth factor 1; intracerebroventricular; intraperitoneal; intravascular; intronic splicing silencer N1; intronic splicing silencer N2; non-steroidal anti-inflammatory drug; scAAV; self-complimentary adeno-associated virus; signal transducer and activator of transcription 5; small nuclear ribonucleoprotein; snRNP; spinal muscular atrophy; subcutaneous; suberoylanilide hydroxamic acid; survival motor neuron; trichostatin A; valproic acid