Investigation of New Morpholino Oligomers to Increase Survival Motor Neuron Protein Levels in Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is an autosomal-recessive childhood motor neuron disease and the main genetic cause of infant mortality. SMA is caused by deletions or mutations in the survival motor neuron 1 (SMN1) gene, which results in SMN protein deficiency. Only one approved drug has recently beco...

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Gepubliceerd in:Int J Mol Sci
Hoofdauteurs: Ramirez, Agnese, Crisafulli, Sebastiano G., Rizzuti, Mafalda, Bresolin, Nereo, Comi, Giacomo P., Corti, Stefania, Nizzardo, Monica
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: MDPI 2018
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Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5796116/
https://ncbi.nlm.nih.gov/pubmed/29316633
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms19010167
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