Antisense-based therapy for the treatment of spinal muscular atrophy
Author:
Affiliation:
1. Isis Pharmaceuticals, Carlsbad, CA 92010
2. Cold Spring Harbor Laboratory, Cold Spring Harbor, NY 11724
Abstract
Publisher
Rockefeller University Press
Subject
Cell Biology
Link
http://rupress.org/jcb/article-pdf/199/1/21/1357614/jcb_201207087.pdf
Reference37 articles.
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3. Early heart failure in the SMNDelta7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery;Bevan;Hum. Mol. Genet.,2010
4. Spinal distribution and metabolism of 2′-O-(2-methoxyethyl)-modified oligonucleotides after intrathecal administration in rats;Butler;Neuroscience.,2005
5. Disruption of an SF2/ASF-dependent exonic splicing enhancer in SMN2 causes spinal muscular atrophy in the absence of SMN1;Cartegni;Nat. Genet.,2002
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