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3. Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissue

4. Complete sequencing of the SMN2 gene in SMA patients detects SMN gene deletion junctions and variants in SMN2 that modify the SMA phenotype

5. Astrocytes from familial and sporadic ALS patients are toxic to motor neurons

9. Linkage mapping of the spinal muscular atrophy gene

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