A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse

Spinal muscular atrophy (SMA) is an autosomal-recessive disorder characterized by α-motor neuron loss in the spinal cord anterior horn. SMA results from deletion or mutation of the Survival Motor Neuron 1 gene (SMN1) and retention of SMN2. A single nucleotide difference between SMN1 and SMN2 results...

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Auteurs principaux: Porensky, Paul N., Mitrpant, Chalermchai, McGovern, Vicki L., Bevan, Adam K., Foust, Kevin D., Kaspar, Brain K., Wilton, Stephen D., Burghes, Arthur H.M.
Format: Artigo
Langue:Inglês
Publié: Oxford University Press 2012
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Articles
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC3298284/
https://ncbi.nlm.nih.gov/pubmed/22186025
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddr600
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