Efficient SMN Rescue following Subcutaneous Tricyclo-DNA Antisense Oligonucleotide Treatment

Efficient SMN Rescue following Subcutaneous Tricyclo-DNA Antisense Oligonucleotide Treatment

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....

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Bibliographic Details
Main Authors: Valérie Robin, Graziella Griffith, John-Paul L. Carter, Christian J. Leumann, Luis Garcia, Aurélie Goyenvalle
Format: Article
Language:English
Published: Elsevier 2017-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
splice switching
spinal muscular atrophy
antisense oligonucleotide
tricyclo-DNA
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253117301385

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http://www.sciencedirect.com/science/article/pii/S2162253117301385

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