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

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

RNA-binding proteins in microsatellite expansion disorders: Mediators of RNA toxicity

(2012) Gloria V. Echeverria et al.   BRAIN RESEARCH

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

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

Structure, function and targeting of human telomere RNA

(2012) Yan Xu et al.   METHODS

Targeting nuclear RNA for in vivo correction of myotonic dystrophy

(2012) Thurman M. Wheeler et al.   NATURE

From dynamic combinatorial ‘hit’ to lead: in vitro and in vivo activity of compounds targeting the pathogenic RNAs that cause myotonic dystrophy

(2012) Leslie O. Ofori et al.   NUCLEIC ACIDS RESEARCH

The four Zn fingers of MBNL1 provide a flexible platform for recognition of its RNA binding elements

(2011) Danielle Cass et al.   BMC MOLECULAR BIOLOGY

MBNL1-RNA Recognition: Contributions of MBNL1 Sequence and RNA Conformation

(2011) Yuan Fu et al.   CHEMBIOCHEM

Kinetic efficiency: the missing metric for enhancing compound quality?

(2011) Geoffrey A. Holdgate et al.   DRUG DISCOVERY TODAY

Therapeutics development in myotonic dystrophy type 1

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

Discovery of selective bioactive small molecules by targeting an RNA dynamic ensemble

(2011) Andrew C Stelzer et al.   Nature Chemical Biology

A sequence-specific threading tetra-intercalator with an extremely slow dissociation rate constant

(2011) Garen G. Holman et al.   Nature Chemistry

New function for the RNA helicase p68/DDX5 as a modifier of MBNL1 activity on expanded CUG repeats

(2011) François-Xavier Laurent 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

Single-molecule analysis of Mss116-mediated group II intron folding

(2010) Krishanthi S. Karunatilaka et al.   NATURE

RNA and Disease

(2009) Thomas A. Cooper et al.   CELL

RNA’s coming of age as a drug target

(2009) Quentin Vicens   JOURNAL OF INCLUSION PHENOMENA AND MACROCYCLIC CHEMISTRY

Single-Molecule Imaging of anin Vitro-Evolved RNA Aptamer Reveals Homogeneous Ligand Binding Kinetics

(2009) Mark P. Elenko 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

Single-Molecule Analysis Reveals Differential Effect of ssDNA-Binding Proteins on DNA Translocation by XPD Helicase

(2009) Masayoshi Honda et al.   MOLECULAR CELL

Proteins and proteomics: life on the surface

(2009) Nathan Blow   NATURE METHODS

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

Fluorescence quenching of TMR by guanosine in oligonucleotides

(2009) Peng Qu et al.   Science in China. Series B, Chemistry

Mechanisms of RNA-mediated Disease

(2008) Jason R. O'Rourke et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Structural insights into RNA recognition by the alternative-splicing regulator muscleblind-like MBNL1

(2008) Marianna Teplova et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY