Modifications to toxic CUG RNAs induce structural stability, rescue mis-splicing in a myotonic dystrophy cell model and reduce toxicity in a myotonic dystrophy zebrafish model

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

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

Structure of the Myotonic Dystrophy Type 2 RNA and Designed Small Molecules That Reduce Toxicity

(2013) Jessica L. Childs-Disney et al.   ACS Chemical Biology

Transcriptional changes and developmental abnormalities in a zebrafish model of myotonic dystrophy type 1

(2013) P. K. Todd et al.   Disease Models & Mechanisms

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

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

Regulation of small RNA stability: methylation and beyond

(2012) Lijuan Ji et al.   CELL RESEARCH

Combinatorial Mutagenesis of MBNL1 Zinc Fingers Elucidates Distinct Classes of Regulatory Events

(2012) J. Purcell et al.   MOLECULAR AND CELLULAR BIOLOGY

Targeting nuclear RNA for in vivo correction of myotonic dystrophy

(2012) Thurman M. Wheeler et al.   NATURE

Overview of theCCP4 suite and current developments

(2011) Martyn D. Winn et al.   ACTA CRYSTALLOGRAPHICA SECTION D-BIOLOGICAL CRYSTALLOGRAPHY

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

Refinement of the Cornell et al. Nucleic Acids Force Field Based on Reference Quantum Chemical Calculations of Glycosidic Torsion Profiles

(2011) Marie Zgarbová et al.   Journal of Chemical Theory and Computation

Expanded GGGGCC Hexanucleotide Repeat in Noncoding Region of C9ORF72 Causes Chromosome 9p-Linked FTD and ALS

(2011) Mariely DeJesus-Hernandez et al.   NEURON

Crystal structures of CGG RNA repeats with implications for fragile X-associated tremor ataxia syndrome

(2011) Agnieszka Kiliszek et al.   NUCLEIC ACIDS RESEARCH

Features and development ofCoot

(2010) P. Emsley et al.   ACTA CRYSTALLOGRAPHICA SECTION D-BIOLOGICAL CRYSTALLOGRAPHY

Zebrafish models of collagen VI-related myopathies

(2010) W.R. Telfer et al.   HUMAN MOLECULAR GENETICS

Zebrafish MTMR14 is required for excitation–contraction coupling, developmental motor function and the regulation of autophagy

(2010) J.J. Dowling et al.   HUMAN MOLECULAR GENETICS

Aberrant alternative splicing and extracellular matrix gene expression in mouse models of myotonic dystrophy

(2010) Hongqing Du et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

MBNL1 binds GC motifs embedded in pyrimidines to regulate alternative splicing

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

Pathogenic mechanisms of myotonic dystrophy

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

PLUMED: A portable plugin for free-energy calculations with molecular dynamics

(2009) Massimiliano Bonomi et al.   COMPUTER PHYSICS COMMUNICATIONS

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

(2009) Agnieszka Kiliszek et al.   NUCLEIC ACIDS RESEARCH

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

RNA Gain-of-Function in Spinocerebellar Ataxia Type 8

(2009) Randy S. Daughters et al.   PLoS Genetics

Mechanisms of RNA-mediated Disease

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

GROMACS 4:  Algorithms for Highly Efficient, Load-Balanced, and Scalable Molecular Simulation

(2008) Berk Hess et al.   Journal of Chemical Theory and Computation

3DNA: a versatile, integrated software system for the analysis, rebuilding and visualization of three-dimensional nucleic-acid structures

(2008) Xiang-Jun Lu et al.   Nature Protocols

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

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