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Restoration of Full-Length SMN Promoted by Adenoviral Vectors Expressing RNA Antisense Oligonucleotides Embedded in U7 snRNAs

BACKGROUND: Spinal Muscular Atrophy (SMA) is an autosomal recessive disease that leads to specific loss of motor neurons. It is caused by deletions or mutations of the survival of motor neuron 1 gene (SMN1). The remaining copy of the gene, SMN2, generates only low levels of the SMN protein due to a...

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Bibliografske podrobnosti
Main Authors: Geib, Till, Hertel, Klemens J.
Format: Artigo
Jezik:Inglês
Izdano: Public Library of Science 2009
Teme:
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC2781471/
https://ncbi.nlm.nih.gov/pubmed/19997596
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0008204
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