Efficient SMN Rescue following Subcutaneous Tricyclo-DNA Antisense Oligonucleotide Treatment

• Auteurs principaux: Valérie Robin (Auteur), Graziella Griffith (Auteur), John-Paul L. Carter (Auteur), Christian J. Leumann (Auteur), Luis Garcia (Auteur), Aurélie Goyenvalle (Auteur)
• Format: Livre
• Publié: Elsevier, 2017-06-01T00:00:00Z.
• Sujets: article
• Accès en ligne: Connect to this object online.

Spinal muscular atrophy (SMA) is a recessive disease caused by mutations in the SMN1 gene, which encodes the protein survival motor neuron (SMN), whose absence dramatically affects the survival of motor neurons. In humans, the severity of the disease is lessened by the presence of a gene copy, SMN2....