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

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

Myotonic dystrophy mouse models: towards rational therapy development

(2011) Mário Gomes-Pereira et al.   TRENDS IN MOLECULAR MEDICINE

Drug–target residence time: critical information for lead optimization

(2010) Hao Lu et al.   CURRENT OPINION IN CHEMICAL BIOLOGY

Strategies for Recognition of Stem−Loop RNA Structures by Synthetic Ligands: Application to the HIV-1 Frameshift Stimulatory Sequence

(2010) Prakash B. Palde et al.   JOURNAL OF MEDICINAL CHEMISTRY

DNA Minor Groove Induced Dimerization of Heterocyclic Cations: Compound Structure, Binding Affinity, and Specificity for a TTAA Site

(2010) Manoj Munde et al.   JOURNAL OF MOLECULAR BIOLOGY

Molecular modeling study of intercalation complexes of tricyclic carboxamides with d(CCGGCGCCGG)2 and d(CGCGAATTCGCG)2

(2010) Athanasia Varvaresou et al.   JOURNAL OF MOLECULAR MODELING

Grading the commercial optical biosensor literature-Class of 2008: ‘The Mighty Binders’

(2010) Rebecca L. Rich et al.   JOURNAL OF MOLECULAR RECOGNITION

Atomic resolution structure of CAG RNA repeats: structural insights and implications for the trinucleotide repeat expansion diseases

(2010) Agnieszka Kiliszek et al.   NUCLEIC ACIDS RESEARCH

Rational and Modular Design of Potent Ligands Targeting the RNA That Causes Myotonic Dystrophy 2

(2009) Melissa M. Lee et al.   ACS Chemical Biology

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

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

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

(2009) T. M. Wheeler et al.   SCIENCE

A global benchmark study using affinity-based biosensors

(2008) Rebecca L. Rich et al.   ANALYTICAL BIOCHEMISTRY

Targeting RNA with Small Molecules

(2008) Jason R. Thomas 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

Myotonic dystrophy: Therapeutic strategies for the future

(2008) Thurman M. Wheeler   Neurotherapeutics