In silico discovery of substituted pyrido[2,3-d]pyrimidines and pentamidine-like compounds with biological activity in myotonic dystrophy models

On the Applicability of Elastic Network Models for the Study of RNA CUG Trinucleotide Repeat Overexpansion

(2016) Àlex L. González et al.   PLoS One

Studying a Drug-like, RNA-Focused Small Molecule Library Identifies Compounds That Inhibit RNA Toxicity in Myotonic Dystrophy

(2015) Suzanne G. Rzuczek et al.   ACS Chemical Biology

Increased autophagy and apoptosis contribute to muscle atrophy in a myotonic dystrophy type 1 Drosophila model

(2015) A. Bargiela et al.   Disease Models & Mechanisms

Computational Investigation of RNA CUG Repeats Responsible for Myotonic Dystrophy 1

(2015) Ilyas Yildirim et al.   Journal of Chemical Theory and Computation

A fly model for the CCUG-repeat expansion of myotonic dystrophy type 2 reveals a novel interaction with MBNL1

(2014) Z. Yu et al.   HUMAN MOLECULAR GENETICS

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

Myotonic Dystrophy

(2014) Charles A. Thornton   NEUROLOGIC CLINICS

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

Reducing Levels of Toxic RNA with Small Molecules

(2013) Leslie A. Coonrod 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

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

Methods to enable the design of bioactive small molecules targeting RNA

(2013) Matthew D. Disney et al.   ORGANIC & BIOMOLECULAR CHEMISTRY

LigandRNA: computational predictor of RNA-ligand interactions

(2013) A. Philips et al.   RNA

Rationally Designed Small Molecules Targeting the RNA That Causes Myotonic Dystrophy Type 1 Are Potently Bioactive

(2012) Jessica L. Childs-Disney et al.   ACS Chemical Biology

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

Utilizing the GAAA Tetraloop/Receptor To Facilitate Crystal Packing and Determination of the Structure of a CUG RNA Helix

(2012) Leslie A. Coonrod et al.   BIOCHEMISTRY

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

Structural dynamics of double-helical RNAs composed of CUG/CUG- and CUG/CGG-repeats

(2012) Jevgenia Tamjar et al.   JOURNAL OF BIOMOLECULAR STRUCTURE & DYNAMICS

Novel Insights of Structure-Based Modeling for RNA-Targeted Drug Discovery

(2012) Lu Chen et al.   Journal of Chemical Information and Modeling

Druggability Assessment of Allosteric Proteins by Dynamics Simulations in the Presence of Probe Molecules

(2012) Ahmet Bakan et al.   Journal of Chemical Theory and Computation

Design of a Bioactive Small Molecule That Targets the Myotonic Dystrophy Type 1 RNA via an RNA Motif–Ligand Database and Chemical Similarity Searching

(2012) Raman Parkesh et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Identifying the preferred RNA motifs and chemotypes that interact by probing millions of combinations

(2012) Tuan Tran et al.   Nature Communications

Recent Advances in Developing Small Molecules Targeting RNA

(2011) Lirui Guan 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

ProDy: Protein Dynamics Inferred from Theory and Experiments

(2011) A. Bakan et al.   BIOINFORMATICS

Understanding RNA Flexibility Using Explicit Solvent Simulations: The Ribosomal and Group I Intron Reverse Kink-Turn Motifs

(2011) Petr Sklenovský et al.   Journal of Chemical Theory and Computation

Selective inhibition of MBNL1–CCUG interaction by small molecules toward potential therapeutic agents for myotonic dystrophy type 2 (DM2) †

(2011) Chun-Ho Wong et al.   NUCLEIC ACIDS RESEARCH

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

Pre-existing soft modes of motion uniquely defined by native contact topology facilitate ligand binding to proteins

(2011) Lidio Meireles et al.   PROTEIN SCIENCE

Large-Scale Systematic Analysis of 2D Fingerprint Methods and Parameters to Improve Virtual Screening Enrichments

(2010) Madhavi Sastry et al.   Journal of Chemical Information and Modeling

Analysis and comparison of 2D fingerprints: Insights into database screening performance using eight fingerprint methods

(2010) Jianxin Duan et al.   JOURNAL OF MOLECULAR GRAPHICS & MODELLING

CHARMM: The biomolecular simulation program

(2009) B. R. Brooks et al.   JOURNAL OF COMPUTATIONAL CHEMISTRY

Controlling the Specificity of Modularly Assembled Small Molecules for RNA via Ligand Module Spacing: Targeting the RNAs That Cause Myotonic Muscular Dystrophy

(2009) Melissa M. Lee et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

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

Interactive exploration of chemical space with Scaffold Hunter

(2009) Stefan Wetzel et al.   Nature Chemical Biology

Structural insights into CUG repeats containing the ‘stretched U–U wobble’: implications for myotonic dystrophy

(2009) Agnieszka Kiliszek et al.   NUCLEIC ACIDS RESEARCH

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