Augmenting the SMN Protein to Treat Infantile Spinal Muscular Atrophy

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Augmenting the SMN Protein to Treat Infantile Spinal Muscular Atrophy Jeong-Ki Kim, Umrao R. Monani Neuron Volume 97, Issue 5, Pages 1001-1003 (March 2018) DOI: 10.1016/j.neuron.2018.02.009 Copyright © 2018 Elsevier Inc. Terms and Conditions

Figure 1 Strategies to Restore SMN as a Means to Treat SMA SMN repletion is effected either by using an antisense moiety such as Nusinersen or by transducing cells with an AAV9 vector harboring a copy of the SMN1 gene. Each approach raises SMN in the cell and rescues/prevents the SMA phenotype. Note: text in gray denotes homozygous mutations in SMN1. Neuron 2018 97, 1001-1003DOI: (10.1016/j.neuron.2018.02.009) Copyright © 2018 Elsevier Inc. Terms and Conditions