High-content screening identifies small molecules that remove nuclear foci, affect MBNL distribution and CELF1 protein levels via a PKC-independent pathway in myotonic dystrophy cell lines

Two high-throughput screening assays for aberrant RNA–protein interactions in myotonic dystrophy type 1

(2012) Catherine Z. Chen et al.   ANALYTICAL AND BIOANALYTICAL CHEMISTRY

GSK3β-cyclin D3-CUGBP1-eIF2 pathway in aging and in Myotonic Dystrophy

(2011) Jingling Jin et al.   CELL CYCLE

Myotonic dystrophy, when simple repeats reveal complex pathogenic entities: new findings and future challenges

(2011) G. Sicot et al.   HUMAN MOLECULAR GENETICS

In vivo discovery of a peptide that prevents CUG-RNA hairpin formation and reverses RNA toxicity in myotonic dystrophy models

(2011) A. Garcia-Lopez et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

The NCGC Pharmaceutical Collection: A Comprehensive Resource of Clinically Approved Drugs Enabling Repurposing and Chemical Genomics

(2011) R. Huang et al.   Science Translational Medicine

Triplet Repeat–Derived siRNAs Enhance RNA–Mediated Toxicity in a Drosophila Model for Myotonic Dystrophy

(2011) Zhenming Yu et al.   PLoS Genetics

The Role of Flexibility in the Rational Design of Modularly Assembled Ligands Targeting the RNAs that Cause the Myotonic Dystrophies

(2010) Matthew D. Disney et al.   CHEMBIOCHEM

Expansion of CUG RNA repeats causes stress and inhibition of translation in myotonic dystrophy 1 (DM1) cells

(2010) Claudia Huichalaf et al.   FASEB JOURNAL

Immortalized Skin Fibroblasts Expressing Conditional MyoD as a Renewable and Reliable Source of Converted Human Muscle Cells to Assess Therapeutic Strategies for Muscular Dystrophies: Validation of an Exon-Skipping Approach to Restore Dystrophin in Duchenne Muscular Dystrophy Cells

(2009) Soraya Chaouch et al.   HUMAN GENE THERAPY

PKC inhibition ameliorates the cardiac phenotype in a mouse model of myotonic dystrophy type 1

(2009) Guey-Shin Wang et al.   JOURNAL OF CLINICAL INVESTIGATION

Reduction of the Rate of Protein Translation in Patients with Myotonic Dystrophy 2

(2009) C. Huichalaf et al.   JOURNAL OF NEUROSCIENCE

Rational Design of Ligands Targeting Triplet Repeating Transcripts That Cause RNA Dominant Disease: Application to Myotonic Muscular Dystrophy Type 1 and Spinocerebellar Ataxia Type 3

(2009) Alexei Pushechnikov et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Efficient gene silencing by delivery of locked nucleic acid antisense oligonucleotides, unassisted by transfection reagents

(2009) C. A. Stein et al.   NUCLEIC ACIDS RESEARCH

Identification of Small Molecule and Genetic Modulators of AON-Induced Dystrophin Exon Skipping by High-Throughput Screening

(2009) Debra A. O'Leary et al.   PLoS One

A simple ligand that selectively targets CUG trinucleotide repeats and inhibits MBNL protein binding

(2009) J. F. Arambula et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Pentamidine reverses the splicing defects associated with myotonic dystrophy

(2009) M. B. Warf et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Triplet-repeat oligonucleotide-mediated reversal of RNA toxicity in myotonic dystrophy

(2009) S. A. M. Mulders et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Reversal of RNA Dominance by Displacement of Protein Sequestered on Triplet Repeat RNA

(2009) T. M. Wheeler et al.   SCIENCE

Dynamic Combinatorial Selection of Molecules Capable of Inhibiting the (CUG) Repeat RNA−MBNL1 Interaction In Vitro: Discovery of Lead Compounds Targeting Myotonic Dystrophy (DM1)

(2008) Peter C. Gareiss et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY