Therapeutics development in myotonic dystrophy type 1

Orthopaedic Manifestations of Congenital Myotonic Dystrophy During Childhood and Adolescence

(2011) Federico Canavese et al.   JOURNAL OF PEDIATRIC ORTHOPAEDICS

Morpholinos and their peptide conjugates: Therapeutic promise and challenge for Duchenne muscular dystrophy

(2010) Hong M. Moulton et al.   BIOCHIMICA ET BIOPHYSICA ACTA-BIOMEMBRANES

Brain involvement in myotonic dystrophies: neuroimaging and neuropsychological comparative study in DM1 and DM2

(2010) Vincenzo Romeo et al.   JOURNAL OF NEUROLOGY

Analysis of CUGBP1 Targets Identifies GU-Repeat Sequences That Mediate Rapid mRNA Decay

(2010) B. Rattenbacher et al.   MOLECULAR AND CELLULAR BIOLOGY

Muscling in: Gene therapies for muscular dystrophy target RNA

(2010) Joel R Chamberlain et al.   NATURE MEDICINE

Repeat instability as the basis for human diseases and as a potential target for therapy

(2010) Arturo López Castel et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

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

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

Comparative analysis of brain structure, metabolism, and cognition in myotonic dystrophy 1 and 2

(2010) Y. G. Weber et al.   NEUROLOGY

Mexiletine is an effective antimyotonia treatment in myotonic dystrophy type 1

(2010) E. L. Logigian et al.   NEUROLOGY

Systematic Analysis of Cis-Elements in Unstable mRNAs Demonstrates that CUGBP1 Is a Key Regulator of mRNA Decay in Muscle Cells

(2010) Jerome E. Lee et al.   PLoS One

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

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

Expression of RNA CCUG Repeats Dysregulates Translation and Degradation of Proteins in Myotonic Dystrophy 2 Patients

(2009) Elizabeth Salisbury et al.   AMERICAN JOURNAL OF PATHOLOGY

Psychiatric and cognitive phenotype in children and adolescents with myotonic dystrophy

(2009) Marie Douniol et al.   EUROPEAN CHILD & ADOLESCENT PSYCHIATRY

Progress in therapeutic antisense applications for neuromuscular disorders

(2009) Annemieke Aartsma-Rus et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

Overcoming biological barriers to in vivo efficacy of antisense oligonucleotides

(2009) Paul J. White et al.   EXPERT REVIEWS IN MOLECULAR MEDICINE

PKC inhibition ameliorates the cardiac phenotype in a mouse model of myotonic dystrophy type 1

(2009) Guey-Shin Wang et al.   JOURNAL OF CLINICAL INVESTIGATION

Absence of a differentiation defect in muscle satellite cells from DM2 patients

(2009) Richard Pelletier et al.   NEUROBIOLOGY OF DISEASE

Myotonic disorders

(2009) Ami Mankodi   NEUROLOGY INDIA

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

Paternally inherited case of congenital DM1: Brain MRI and review of literature

(2008) Alfonso Di Costanzo et al.   BRAIN & DEVELOPMENT

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

Conserved GU-Rich Elements Mediate mRNA Decay by Binding to CUG-Binding Protein 1

(2008) Irina A. Vlasova et al.   MOLECULAR CELL

Dehydroepiandrosterone for myotonic dystrophy type 1

(2008) I. Penisson-Besnier et al.   NEUROLOGY

Expanded CTG repeats within the DMPK 3' UTR causes severe skeletal muscle wasting in an inducible mouse model for myotonic dystrophy

(2008) J. P. Orengo et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA