A Potent Inhibitor of Protein Sequestration by Expanded Triplet (CUG) Repeats that Shows Phenotypic Improvements in aDrosophilaModel of Myotonic Dystrophy

Rationally Designed Small Molecules That Target Both the DNA and RNA Causing Myotonic Dystrophy Type 1

(2015) Lien Nguyen et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Small Molecules that Target the Toxic RNA in Myotonic Dystrophy Type 2

(2014) Lien Nguyen et al.   ChemMedChem

Development of aDrosophila melanogasterspliceosensor system forin vivohigh-throughput screening in myotonic dystrophy type 1

(2014) Irma García-Alcover et al.   Disease Models & Mechanisms

Targeting Toxic RNAs that Cause Myotonic Dystrophy Type 1 (DM1) with a Bisamidinium Inhibitor

(2014) Chun-Ho Wong et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Lomofungin and dilomofungin: inhibitors of MBNL1-CUG RNA binding with distinct cellular effects

(2014) Jason W. Hoskins et al.   NUCLEIC ACIDS RESEARCH

MBNL proteins and their target RNAs, interaction and splicing regulation

(2014) Patryk Konieczny et al.   NUCLEIC ACIDS RESEARCH

Features of Modularly Assembled Compounds That Impart Bioactivity Against an RNA Target

(2013) Suzanne G. Rzuczek et al.   ACS Chemical Biology

A Novel CUGexp·MBNL1 Inhibitor with Therapeutic Potential for Myotonic Dystrophy Type 1

(2013) Amin Haghighat Jahromi et al.   ACS Chemical Biology

Novel Drosophila model of myotonic dystrophy type 1: phenotypic characterization and genome-wide view of altered gene expression

(2013) Lucie Picchio et al.   HUMAN MOLECULAR GENETICS

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

(2013) Ami Ketley et al.   HUMAN MOLECULAR GENETICS

Developing Bivalent Ligands to Target CUG Triplet Repeats, the Causative Agent of Myotonic Dystrophy Type 1

(2013) Amin Haghighat Jahromi et al.   JOURNAL OF MEDICINAL CHEMISTRY

Single-molecule study of the CUG repeat–MBNL1 interaction and its inhibition by small molecules

(2013) Amin Haghighat Jahromi et al.   NUCLEIC ACIDS RESEARCH

Induction and reversal of myotonic dystrophy type 1 pre-mRNA splicing defects by small molecules

(2013) Jessica L. Childs-Disney et al.   Nature Communications

Investigating the Binding Mode of an Inhibitor of the MBNL1⋅RNA Complex in Myotonic Dystrophy Type 1 (DM1) Leads to the Unexpected Discovery of a DNA-Selective Binder

(2012) Chun-Ho Wong et al.   CHEMBIOCHEM

Mouse model of muscleblind-like 1 overexpression: skeletal muscle effects and therapeutic promise

(2012) Christopher M. Chamberlain et al.   HUMAN MOLECULAR GENETICS

Structure of the HIV-1 Frameshift Site RNA Bound to a Small Molecule Inhibitor of Viral Replication

(2011) Ryan J. Marcheschi et al.   ACS Chemical Biology

Myotonic Dystrophy Type 1 RNA Crystal Structures Reveal Heterogeneous 1 × 1 Nucleotide UU Internal Loop Conformations

(2011) Amit Kumar et al.   BIOCHEMISTRY

Therapeutics development in myotonic dystrophy type 1

(2011) Erin Pennock Foff et al.   MUSCLE & NERVE

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

Perspectives on gene therapy in myotonic dystrophy type 1

(2010) Jonathan J. Magaña et al.   JOURNAL OF NEUROSCIENCE RESEARCH

Pathogenic mechanisms of myotonic dystrophy

(2009) Johanna E. Lee et al.   BIOCHEMICAL SOCIETY TRANSACTIONS

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

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

(2009) T. M. Wheeler et al.   SCIENCE

Cu-Catalyzed Azide−Alkyne Cycloaddition

(2008) Morten Meldal et al.   CHEMICAL REVIEWS

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

Genetic and Chemical Modifiers of a CUG Toxicity Model in Drosophila

(2008) Amparo Garcia-Lopez et al.   PLoS One