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Augmenting the SMN Protein to Treat Infantile Spinal Muscular Atrophy

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Presentation on theme: "Augmenting the SMN Protein to Treat Infantile Spinal Muscular Atrophy"— Presentation transcript:

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

2 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  , DOI: ( /j.neuron ) Copyright © 2018 Elsevier Inc. Terms and Conditions

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