Improved Antisense Oligonucleotide Design to Suppress Aberrant SMN2 Gene Transcript Processing: Towards a Treatment for Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is caused by loss of the Survival Motor Neuron 1 (SMN1) gene, resulting in reduced SMN protein. Humans possess the additional SMN2 gene (or genes) that does produce low level of full length SMN, but cannot adequately compensate for loss of SMN1 due to aberrant splicing....

Full description

Saved in:
Bibliographic Details
Main Authors: Mitrpant, Chalermchai, Porensky, Paul, Zhou, Haiyan, Price, Loren, Muntoni, Francesco, Fletcher, Sue, Wilton, Steve D., Burghes, Arthur H. M.
Format: Artigo
Language:Inglês
Published: Public Library of Science 2013
Subjects:
Research Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC3632594/
https://ncbi.nlm.nih.gov/pubmed/23630626
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0062114
Tags: Add Tag
No Tags, Be the first to tag this record!

Similar Items