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...

Descripción completa

Guardado en:
Detalles Bibliográficos
Autores principales: Agnese Ramirez, Sebastiano G. Crisafulli, Mafalda Rizzuti, Nereo Bresolin, Giacomo P. Comi, Stefania Corti, Monica Nizzardo
Formato: Artigo
Lenguaje:Inglês
Publicado: MDPI AG 2018-01-01
Colección:International Journal of Molecular Sciences
Materias:
spinal muscular atrophy
morpholino
therapy
Acceso en línea:http://www.mdpi.com/1422-0067/19/1/167
Etiquetas: Agregar Etiqueta
Sin Etiquetas, Sea el primero en etiquetar este registro!

Ejemplares similares