SMN1 encodes the survival motor neuron protein, which is essential for motor neuron maintenance and function 1. The primary function involves catalyzing the assembly of small nuclear ribonucleoproteins (snRNPs), critical components of the spliceosome that enable proper splicing of cellular pre-mRNAs. SMN1 works in conjunction with SMN2, a paralogous gene that produces reduced levels of functional SMN protein due to alternative splicing caused by a single nucleotide difference (C-to-T transition) that promotes exon 7 skipping 2. This splicing defect explains why SMN2 cannot fully compensate for SMN1 loss. SMN1 mutations cause spinal muscular atrophy (SMA), an autosomal recessive neurodegenerative disorder characterized by spinal motor neuron degeneration, skeletal muscle atrophy, and generalized weakness 34. Disease severity inversely correlates with SMN2 gene copy number, as SMN2 produces only a fraction of functional SMN protein compared to SMN1 4. SMA affects approximately 1-2 per 100,000 persons with an incidence of 1 in 10,000 live births 5. Clinically, gene therapy using AAV9 vectors to deliver functional SMN1 has shown promise, with onasemnogene abeparvovec approved for SMA treatment, demonstrating improved motor function and survival when administered early 16.