| SMA | Spinal Muscular Atrophy |
| SMN | Survival Motor Neuron |
| ASO | Antisense oligonucleotide |
| MO | Morpholino |
| IPSCs | Induced pluripotent stem cells |
| MN | Motor neuron |
| SCR | Scrambled |
| ICV | Intracerebroventricular |
| SC | Subcutaneous |
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