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...

Full description

Saved in:
Bibliographic Details
Main Authors: Agnese Ramirez, Sebastiano G. Crisafulli, Mafalda Rizzuti, Nereo Bresolin, Giacomo P. Comi, Stefania Corti, Monica Nizzardo
Format: Artigo
Language:Inglês
Published: MDPI AG 2018-01-01
Series:International Journal of Molecular Sciences
Subjects:
spinal muscular atrophy
morpholino
therapy
Online Access:http://www.mdpi.com/1422-0067/19/1/167
Tags: Add Tag
No Tags, Be the first to tag this record!

Similar Items