Structures of RNA repeats associated with neurological diseases

Pathogenic C9ORF72 Antisense Repeat RNA Forms a Double Helix with Tandem C:C Mismatches

(2016) David W. Dodd et al.   BIOCHEMISTRY

RAN translation—What makes it run?

(2016) Katelyn M. Green et al.   BRAIN RESEARCH

Structural characterization of a dimer of RNA duplexes composed of 8-bromoguanosine modified CGG trinucleotide repeats: a novel architecture of RNA quadruplexes

(2016) Dorota Gudanis et al.   NUCLEIC ACIDS RESEARCH

Crystallographic and Computational Analyses of AUUCU Repeating RNA That Causes Spinocerebellar Ataxia Type 10 (SCA10)

(2015) HaJeung Park et al.   BIOCHEMISTRY

Invited Review: Decoding the pathophysiological mechanisms that underlie RNA dysregulation in neurodegenerative disorders: a review of the current state of the art

(2015) Matthew J. Walsh et al.   NEUROPATHOLOGY AND APPLIED NEUROBIOLOGY

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

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

Molecular mechanisms in DM1 — a focus on foci

(2015) Olof Joakim Pettersson et al.   NUCLEIC ACIDS RESEARCH

The first crystal structures of RNA–PNA duplexes and a PNA-PNA duplex containing mismatches—toward anti-sense therapy against TREDs

(2015) Agnieszka Kiliszek et al.   NUCLEIC ACIDS RESEARCH

Structural Insights Reveal the Dynamics of the Repeating r(CAG) Transcript Found in Huntington’s Disease (HD) and Spinocerebellar Ataxias (SCAs)

(2015) Arpita Tawani et al.   PLoS One

Watson–Crick-like pairs in CCUG repeats: evidence for tautomeric shifts or protonation

(2015) Wojciech Rypniewski et al.   RNA

Mechanisms of toxicity in C9FTLD/ALS

(2014) Tania F. Gendron et al.   ACTA NEUROPATHOLOGICA

Mechanisms of transcriptional dysregulation in repeat expansion disorders

(2014) Matthias Groh et al.   BIOCHEMICAL SOCIETY TRANSACTIONS

RNA–protein interactions in unstable microsatellite diseases

(2014) Apoorva Mohan et al.   BRAIN RESEARCH

Polyglutamine (PolyQ) Diseases: Genetics to Treatments

(2014) Hueng-Chuen Fan et al.   CELL TRANSPLANTATION

Trinucleotide expansion in disease: why is there a length threshold?

(2014) Do-Yup Lee et al.   CURRENT OPINION IN GENETICS & DEVELOPMENT

Peptide nucleic acids: a review on recent patents and technology transfer

(2014) Roberto Gambari   EXPERT OPINION ON THERAPEUTIC PATENTS

Potential therapeutic targets in polyglutamine-mediated diseases

(2014) Masahisa Katsuno et al.   Expert Review of Neurotherapeutics

C9orf72 nucleotide repeat structures initiate molecular cascades of disease

(2014) Aaron R. Haeusler et al.   NATURE

Discovery of a Biomarker and Lead Small Molecules to Target r(GGGGCC)-Associated Defects in c9FTD/ALS

(2014) Zhaoming Su et al.   NEURON

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

(2014) Elaine deLorimier et al.   NUCLEIC ACIDS RESEARCH

Structural studies of CNG repeats

(2014) Agnieszka Kiliszek et al.   NUCLEIC ACIDS RESEARCH

RAN translation and frameshifting as translational challenges at simple repeats of human neurodegenerative disorders

(2014) Marzena Wojciechowska et al.   NUCLEIC ACIDS RESEARCH

Oligonucleotide-based strategies to combat polyglutamine diseases

(2014) A. Fiszer et al.   NUCLEIC ACIDS RESEARCH

MBNL proteins and their target RNAs, interaction and splicing regulation

(2014) Patryk Konieczny et al.   NUCLEIC ACIDS RESEARCH

An introduction to recurrent nucleotide interactions in RNA

(2014) Blake A. Sweeney et al.   Wiley Interdisciplinary Reviews-RNA

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

Role of Context in RNA Structure: Flanking Sequences Reconfigure CAG Motif Folding in Huntingtin Exon 1 Transcripts

(2013) Steven Busan et al.   BIOCHEMISTRY

A Dynamic Structural Model of Expanded RNA CAG Repeats: A Refined X-ray Structure and Computational Investigations Using Molecular Dynamics and Umbrella Sampling Simulations

(2013) Ilyas Yildirim et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Treatment of Type 1 Myotonic Dystrophy by Engineering Site-specific RNA Endonucleases that Target (CUG) n Repeats

(2013) Wenjing Zhang et al.   MOLECULAR THERAPY

The Unstable Repeats—Three Evolving Faces of Neurological Disease

(2013) David L. Nelson et al.   NEURON

Design and Analysis of Effects of Triplet Repeat Oligonucleotides in Cell Models for Myotonic Dystrophy

(2013) Anchel González-Barriga et al.   Molecular Therapy-Nucleic Acids

Trinucleotide Repeats: A Structural Perspective

(2013) Bruno Almeida et al.   Frontiers in Neurology

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

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

Triplet repeats in transcripts: structural insights into RNA toxicity

(2012) Paulina Galka-Marciniak et al.   BIOLOGICAL CHEMISTRY

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

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

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

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

RNA Interference Targeting CUG Repeats in a Mouse Model of Myotonic Dystrophy

(2012) Krzysztof Sobczak et al.   MOLECULAR THERAPY

Targeting nuclear RNA for in vivo correction of myotonic dystrophy

(2012) Thurman M. Wheeler et al.   NATURE

Crystallographic characterization of CCG repeats

(2012) Agnieszka Kiliszek et al.   NUCLEIC ACIDS RESEARCH

RNase H-mediated degradation of toxic RNA in myotonic dystrophy type 1

(2012) J. E. Lee et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

C9orf72 hexanucleotide repeat associated with amyotrophic lateral sclerosis and frontotemporal dementia forms RNA G-quadruplexes

(2012) Pietro Fratta et al.   Scientific Reports

Myotonic Dystrophy Type 1 RNA Crystal Structures Reveal Heterogeneous 1 × 1 Nucleotide UU Internal Loop Conformations

(2011) Amit Kumar et al.   BIOCHEMISTRY

A Crystal Structure of a Model of the Repeating r(CGG) Transcript Found in Fragile X Syndrome

(2011) Amit Kumar et al.   CHEMBIOCHEM

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

(2011) Yuan Fu et al.   CHEMBIOCHEM

Cellular toxicity of expanded RNA repeats: focus on RNA foci

(2011) Marzena Wojciechowska et al.   HUMAN MOLECULAR GENETICS

Repetitive DNA and next-generation sequencing: computational challenges and solutions

(2011) Todd J. Treangen et al.   NATURE REVIEWS GENETICS

Triplet repeat RNA structure and its role as pathogenic agent and therapeutic target

(2011) W. J. Krzyzosiak et al.   NUCLEIC ACIDS RESEARCH

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

(2011) Agnieszka Kiliszek et al.   NUCLEIC ACIDS RESEARCH

Mutant CAG repeats of Huntingtin transcript fold into hairpins, form nuclear foci and are targets for RNA interference

(2011) Mateusz de Mezer et al.   NUCLEIC ACIDS RESEARCH

Spinocerebellar ataxia type 10 – A review

(2011) Hélio A.G. Teive et al.   PARKINSONISM & RELATED DISORDERS

Features and development ofCoot

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

RNAstructure: software for RNA secondary structure prediction and analysis

(2010) Jessica S Reuter et al.   BMC BIOINFORMATICS

Structural Diversity of Triplet Repeat RNAs

(2010) Krzysztof Sobczak et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Repeat expansion disease: progress and puzzles in disease pathogenesis

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

Selective silencing of mutated mRNAs in DM1 by using modified hU7-snRNAs

(2010) Virginie François et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

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

Repeat expansion diseases: when a good RNA turns bad

(2010) Alexa M. Dickson et al.   Wiley Interdisciplinary Reviews-RNA

VARNA: Interactive drawing and editing of the RNA secondary structure

(2009) Kévin Darty et al.   BIOINFORMATICS

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

(2009) Agnieszka Kiliszek et al.   NUCLEIC ACIDS RESEARCH

The quadruplex r(CGG)n destabilizing cationic porphyrin TMPyP4 cooperates with hnRNPs to increase the translation efficiency of fragile X premutation mRNA

(2009) N. Ofer et al.   NUCLEIC ACIDS RESEARCH

Web 3DNA--a web server for the analysis, reconstruction, and visualization of three-dimensional nucleic-acid structures

(2009) G. Zheng 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

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

(2009) T. M. Wheeler et al.   SCIENCE

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

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

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