Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice

Impacts of Dystrophin and Utrophin Domains on Actin Structural Dynamics: Implications for Therapeutic Design

(2012) Ava Yun Lin et al.   JOURNAL OF MOLECULAR BIOLOGY

Ribosomal Protein S25 Dependency Reveals a Common Mechanism for Diverse Internal Ribosome Entry Sites and Ribosome Shunting

(2012) M. I. Hertz et al.   MOLECULAR AND CELLULAR BIOLOGY

Muscle Function Recovery in Golden Retriever Muscular Dystrophy After AAV1-U7 Exon Skipping

(2012) Adeline Vulin et al.   MOLECULAR THERAPY

Becker muscular dystrophy with widespread muscle hypertrophy and a non-sense mutation of exon 2

(2012) N. Witting et al.   NEUROMUSCULAR DISORDERS

The passive mechanical properties of the extensor digitorum longus muscle are compromised in 2- to 20-mo-old mdx mice

(2011) Chady H. Hakim et al.   JOURNAL OF APPLIED PHYSIOLOGY

The Carboxy-Terminal Third of Dystrophin Enhances Actin Binding Activity

(2011) Davin M. Henderson et al.   JOURNAL OF MOLECULAR BIOLOGY

Total RNA sequencing reveals nascent transcription and widespread co-transcriptional splicing in the human brain

(2011) Adam Ameur et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

Dystrophin: More than just the sum of its parts

(2010) Elisabeth Le Rumeur et al.   BIOCHIMICA ET BIOPHYSICA ACTA-PROTEINS AND PROTEOMICS

RNA-targeted splice-correction therapy for neuromuscular disease

(2010) M. J. A. Wood et al.   BRAIN

Translational Regulation of Gene Expression during Conditions of Cell Stress

(2010) Keith A. Spriggs et al.   MOLECULAR CELL

Immortalized Skin Fibroblasts Expressing Conditional MyoD as a Renewable and Reliable Source of Converted Human Muscle Cells to Assess Therapeutic Strategies for Muscular Dystrophies: Validation of an Exon-Skipping Approach to Restore Dystrophin in Duchenne Muscular Dystrophy Cells

(2009) Soraya Chaouch et al.   HUMAN GENE THERAPY

DMDexon 1 truncating point mutations: Amelioration of phenotype by alternative translation initiation in exon 6

(2009) Olga L. Gurvich et al.   HUMAN MUTATION

Mutational spectrum of DMD mutations in dystrophinopathy patients: application of modern diagnostic techniques to a large cohort

(2009) Kevin M. Flanigan et al.   HUMAN MUTATION

Efficient bypass of mutations in dysferlin deficient patient cells by antisense-induced exon skipping

(2009) Nicolas Wein et al.   HUMAN MUTATION

Dystrophin is a microtubule-associated protein

(2009) Kurt W. Prins et al.   JOURNAL OF CELL BIOLOGY

Preclinical drug trials in themdxmouse: Assessment of reliable and sensitive outcome measures

(2009) Christopher F. Spurney et al.   MUSCLE & NERVE

DMD Trp3X nonsense mutation associated with a founder effect in North American families with mild Becker muscular dystrophy

(2009) Kevin M. Flanigan et al.   NEUROMUSCULAR DISORDERS

Upstream open reading frames cause widespread reduction of protein expression and are polymorphic among humans

(2009) S. E. Calvo et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A novel custom high density-comparative genomic hybridization array detects common rearrangements as well as deep intronic mutations in dystrophinopathies

(2008) Matteo Bovolenta et al.   BMC GENOMICS

IRES-Mediated Translation of Utrophin A Is Enhanced by Glucocorticoid Treatment in Skeletal Muscle Cells

(2008) Pedro Miura et al.   PLoS One