Dantrolene Enhances Antisense-Mediated Exon Skipping in Human and Mouse Models of Duchenne Muscular Dystrophy

Exon-skipping drug pulls ahead in muscular dystrophy field

(2012) Alisa Opar   NATURE MEDICINE

Ryanodine Receptor Oxidation Causes Intracellular Calcium Leak and Muscle Weakness in Aging

(2011) Daniel C. Andersson et al.   Cell Metabolism

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

(2011) Sebahattin Cirak et al.   LANCET

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

(2011) Nathalie M. Goemans et al.   NEW ENGLAND JOURNAL OF MEDICINE

Genetic therapies for RNA mis-splicing diseases

(2011) Suzan M. Hammond et al.   TRENDS IN GENETICS

Chemical treatment enhances skipping of a mutated exon in the dystrophin gene

(2011) Atsushi Nishida et al.   Nature Communications

Recognizing and managing a malignant hyperthermia crisis: guidelines from the European Malignant Hyperthermia Group

(2010) K.P.E. Glahn et al.   BRITISH JOURNAL OF ANAESTHESIA

Diaphragm rescue alone prevents heart dysfunction in dystrophic mice

(2010) Alastair Crisp et al.   HUMAN MOLECULAR GENETICS

Guanine Analogues Enhance Antisense Oligonucleotide-induced Exon Skipping in Dystrophin Gene In Vitro and In Vivo

(2010) Yihong Hu et al.   MOLECULAR THERAPY

One-year Treatment of Morpholino Antisense Oligomer Improves Skeletal and Cardiac Muscle Functions in Dystrophic mdx Mice

(2010) Bo Wu et al.   MOLECULAR THERAPY

Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs

(2009) Toshifumi Yokota et al.   ANNALS OF NEUROLOGY

Dose-dependent restoration of dystrophin expression in cardiac muscle of dystrophic mice by systemically delivered morpholino

(2009) B Wu et al.   GENE THERAPY

Dosing Regimen Has a Significant Impact on the Efficiency of Morpholino Oligomer-Induced Exon Skipping in mdx Mice

(2009) Alberto Malerba et al.   HUMAN GENE THERAPY

Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations

(2009) Annemieke Aartsma-Rus et al.   HUMAN MUTATION

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

(2009) Maria Kinali et al.   LANCET NEUROLOGY

Hypernitrosylated ryanodine receptor calcium release channels are leaky in dystrophic muscle

(2009) Andrew M Bellinger et al.   NATURE MEDICINE

Identification of Small Molecule and Genetic Modulators of AON-Induced Dystrophin Exon Skipping by High-Throughput Screening

(2009) Debra A. O'Leary et al.   PLoS One

The influence of calcium signaling on the regulation of alternative splicing

(2008) Joachim Krebs   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR CELL RESEARCH

Reference genes for normalization of gene expression studies in human osteoarthritic articular cartilage

(2008) Manuel Pombo-Suarez et al.   BMC MOLECULAR BIOLOGY

Cell-lineage regulated myogenesis for dystrophin replacement: a novel therapeutic approach for treatment of muscular dystrophy

(2008) E. Kimura et al.   HUMAN MOLECULAR GENETICS

Follow-up of three patients with a large in-frame deletion of exons 45–55 in the Duchenne muscular dystrophy (DMD) gene

(2008) Akinori Nakamura et al.   JOURNAL OF CLINICAL NEUROSCIENCE

Generation of human induced pluripotent stem cells from dermal fibroblasts

(2008) W. E. Lowry et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA