Perspectives on gene therapy in myotonic dystrophy type 1
Author:
Publisher
Wiley
Subject
Cellular and Molecular Neuroscience
Link
http://onlinelibrary.wiley.com/wol1/doi/10.1002/jnr.22551/fullpdf
Reference124 articles.
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3. A cell-based assay for aggregation inhibitors as therapeutics of polyglutamine-repeat disease and validation in Drosophila;Apostol;Proc Natl Acad Sci U S A,2003
4. A simple ligand that selectively targets CUG trinucleotide repeats and inhibits MBNL protein binding;Arambula;Proc Natl Acad Sci U S A,2009
5. Intergenerational stability of the myotonic dystrophy protomutation;Barcelo;Hum Mol Genet,1993
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