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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Hauptverfasser: Agnese Ramirez, Sebastiano G. Crisafulli, Mafalda Rizzuti, Nereo Bresolin, Giacomo P. Comi, Stefania Corti, Monica Nizzardo
Format: Artigo
Sprache:Inglês
Veröffentlicht: MDPI AG 2018-01-01
Schriftenreihe:International Journal of Molecular Sciences
Schlagworte:
spinal muscular atrophy
morpholino
therapy
Online Zugang:http://www.mdpi.com/1422-0067/19/1/167
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