Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment

Corinne Betts, Amer F. Saleh, Andrey Arzumanov, +5

Molecular Therapy — Nucleic Acids

Brain involvement in muscular dystrophies with defective dystroglycan glycosylation

Emma Clement, Eugenio Mercuri, Caroline Godfrey, +19

Annals of Neurology

<i>Fukutin</i> gene mutations in steroid‐responsive limb girdle muscular dystrophy

Caroline Godfrey, Diana M. Escolar, Martin Brockington, +11

Annals of Neurology

Expression Analysis in Multiple Muscle Groups and Serum Reveals Complexity in the MicroRNA Transcriptome of the mdx Mouse with Implications for Therapy

Thomas C. Roberts, K. Emelie M. Blomberg, Graham McClorey, +6

Molecular Therapy — Nucleic Acids

Muscle TissueMuscle FunctionEngineeringCellular PhysiologyTranscriptional Regulation +16

How much dystrophin is enough: the physiological consequences of different levels of dystrophin in the<i>mdx</i>mouse

Caroline Godfrey, S. Muses, Graham McClorey, +11

Human Molecular Genetics

Muscle FunctionEngineeringMammalian PhysiologyCytoskeletonMechanotransduction +17

A Comparative Study of α‐Dystroglycan Glycosylation in Dystroglycanopathies Suggests that the Hypoglycosylation of α‐Dystroglycan Does Not Consistently Correlate with Clinical Severity

C. Jimenez‐Mallebrera, Silvia Torelli, Lucy Feng, +16

Brain Pathology

Peptide-mediated Cell and In Vivo Delivery of Antisense Oligonucleotides and siRNA

Peter Järver, Thibault Coursindel, Samir EL Andaloussi, +3

Molecular Therapy — Nucleic Acids

Extracellular microRNAs are dynamic non-vesicular biomarkers of muscle turnover

Thomas C. Roberts, Caroline Godfrey, Graham McClorey, +7

Nucleic Acids Research

Muscle FunctionSerum Mirna ProfilingPathologyCellular PhysiologyRegenerative Medicine +14

Mild POMGnT1 Mutations Underlie a Novel Limb-Girdle Muscular Dystrophy Variant

Emma Clement, Caroline Godfrey, Jenny Tan, +11

Archives of Neurology

Comprehensive RNA-Sequencing Analysis in Serum and Muscle Reveals Novel Small RNA Signatures with Biomarker Potential for DMD

Anna Coenen-Stass, Helena Sork, Sole Gatto, +11

Molecular Therapy — Nucleic Acids

EngineeringPathologyTranscriptomics TechnologyLong Non-coding RnaMolecular Diagnostics +14