A Dimeric 2,9‐Diamino‐1,10‐phenanthroline Derivative Improves Alternative Splicing in Myotonic Dystrophy Type 1 Cell and Mouse Models

Inforna 2.0: A Platform for the Sequence-Based Design of Small Molecules Targeting Structured RNAs

(2016) Matthew D. Disney et al.   ACS Chemical Biology

A Ligand That Targets CUG Trinucleotide Repeats

(2016) Jinxing Li et al.   CHEMISTRY-A EUROPEAN JOURNAL

Naphthyridine-Benzoazaquinolone: Evaluation of a Tricyclic System for the Binding to (CAG)nRepeat DNA and RNA

(2016) Jinxing Li  et al.   Chemistry-An Asian Journal

Precise small-molecule recognition of a toxic CUG RNA repeat expansion

(2016) Suzanne G Rzuczek et al.   Nature Chemical Biology

2-Aminophenanthroline dimer stabilized the C–C mismatched duplex DNA

(2015) Jinxing Li et al.   BIOORGANIC & MEDICINAL CHEMISTRY

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

Short antisense-locked nucleic acids (all-LNAs) correct alternative splicing abnormalities in myotonic dystrophy

(2015) Agnieszka Wojtkowiak-Szlachcic et al.   NUCLEIC ACIDS RESEARCH

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

Small molecule kinase inhibitors alleviate different molecular features of myotonic dystrophy type 1

(2014) Marzena Wojciechowska et al.   RNA Biology

Recognition of Chelerythrine to Human Telomeric DNA and RNA G-quadruplexes

(2014) Li-Ping Bai et al.   Scientific Reports

Reducing Levels of Toxic RNA with Small Molecules

(2013) Leslie A. Coonrod et al.   ACS Chemical Biology

Splicing biomarkers of disease severity in myotonic dystrophy

(2013) Masayuki Nakamori et al.   ANNALS OF NEUROLOGY

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

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

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

Rational Design of Bioactive, Modularly Assembled Aminoglycosides Targeting the RNA that Causes Myotonic Dystrophy Type 1

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

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

A small molecule regulates hairpin structures in d(CGG) trinucleotide repeats

(2012) Masaki Hagihara et al.   BIOORGANIC & MEDICINAL CHEMISTRY LETTERS

Transcriptome-wide Regulation of Pre-mRNA Splicing and mRNA Localization by Muscleblind Proteins

(2012) Eric T. Wang et al.   CELL

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

Recent Advances in Developing Small Molecules Targeting RNA

(2011) Lirui Guan et al.   ACS Chemical Biology

Small Molecule Modulates Hairpin Structures in CAG Trinucleotide Repeats

(2011) Masaki Hagihara et al.   CHEMBIOCHEM

Stabilization of Expanded (CTG)•(CAG) Repeats by Antisense Oligonucleotides

(2011) Masayuki Nakamori et al.   MOLECULAR THERAPY

Bidirectional transcription stimulates expansion and contraction of expanded (CTG)•(CAG) repeats

(2010) Masayuki Nakamori et al.   HUMAN MOLECULAR GENETICS

Repeat expansion disease: progress and puzzles in disease pathogenesis

(2010) Albert R. La Spada et al.   NATURE REVIEWS GENETICS

MBNL1 binds GC motifs embedded in pyrimidines to regulate alternative splicing

(2010) Emily S. Goers et al.   NUCLEIC ACIDS RESEARCH

Recognition of Mismatched Base Pairs in DNA

(2009) Kazuhiko Nakatani   BULLETIN OF THE CHEMICAL SOCIETY OF JAPAN

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

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

Targeting RNA with Small Molecules

(2008) Jason R. Thomas et al.   CHEMICAL REVIEWS