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Therapeutic advances in 5q-linked spinal muscular atrophy

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Academic year: 2019

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Figure 1. Genetic cause of spinal muscular atrophy (SMA). A) In normal individuals, SMN1 produces 100% of full-length SMN  protein
Figure 2. Therapeutic strategies for spinal muscular atrophy (SMA). A) ASO/nusinersen binds to the ISS-N1 (intronic splicing  silencer N1) in the intron downstream of exon 7 of the  SMN2  transcript increasing exon 7 inclusion in  SMN2 -mRNA and

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