In Silico Screening Based on Predictive Algorithms as a Design Tool for Exon Skipping Oligonucleotides in Duchenne Muscular Dystrophy

New developments in exon skipping and splice modulation therapies for neuromuscular diseases

(2014) Aleksander Touznik et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA

(2014) C. Long et al.   SCIENCE

Spinal muscular atrophy: An update on therapeutic progress

(2013) Joonbae Seo et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

Rescue of cardiomyopathy through U7snRNA-mediated exon skipping inMybpc3-targeted knock-in mice

(2013) Christina Gedicke-Hornung et al.   EMBO Molecular Medicine

Exon Skipping as a Therapeutic Strategy Applied to an RYR1 Mutation with Pseudo-Exon Inclusion Causing a Severe Core Myopathy

(2013) John Rendu et al.   HUMAN GENE THERAPY

Highly efficient in vivo delivery of PMO into regenerating myotubes and rescue in laminin-α2 chain-null congenital muscular dystrophy mice

(2013) Yoshitsugu Aoki et al.   HUMAN MOLECULAR GENETICS

In Vitro Correction of a Pseudoexon-Generating Deep Intronic Mutation in LGMD2A by Antisense Oligonucleotides and Modified Small Nuclear RNAs

(2013) Lorea Blázquez et al.   HUMAN MUTATION

Regulation of alternative splicing by local histone modifications: potential roles for RNA-guided mechanisms

(2013) H.-L. Zhou et al.   NUCLEIC ACIDS RESEARCH

Cellular trafficking determines the exon skipping activity of Pip6a-PMO in mdx skeletal and cardiac muscle cells

(2013) T. Lehto et al.   NUCLEIC ACIDS RESEARCH

Context Dependent Effects of Chimeric Peptide Morpholino Conjugates Contribute to Dystrophin Exon-skipping Efficiency

(2013) HaiFang Yin et al.   Molecular Therapy-Nucleic Acids

Inhibition of IL-1 Signaling by Antisense Oligonucleotide-mediated Exon Skipping of IL-1 Receptor Accessory Protein (IL-1RAcP)

(2013) A Seda Yılmaz-Eliş et al.   Molecular Therapy-Nucleic Acids

Categorization of 77 dystrophin exons into 5 groups by a decision tree using indexes of splicing regulatory factors as decision markers

(2012) Rusdy Malueka et al.   BMC GENETICS

Antisense Oligonucleotide-Mediated Exon Skipping for Duchenne Muscular Dystrophy: Progress and Challenges

(2012) Virginia Arechavala-Gomeza et al.   CURRENT GENE THERAPY

A Prospective Study in the Rational Design of Efficient Antisense Oligonucleotides for Exon Skipping in the DMD Gene

(2012) Zacharias Aloysius Dwi Pramono et al.   HUMAN GENE THERAPY

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

(2012) Adeline Vulin et al.   MOLECULAR THERAPY

Global or local? Predicting secondary structure and accessibility in mRNAs

(2012) Sita J. Lange et al.   NUCLEIC ACIDS RESEARCH

Differential GC Content between Exons and Introns Establishes Distinct Strategies of Splice-Site Recognition

(2012) Maayan Amit et al.   Cell Reports

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

(2012) Corinne Betts et al.   Molecular Therapy-Nucleic Acids

ViennaRNA Package 2.0

(2011) Ronny Lorenz et al.   Algorithms for Molecular Biology

Restoring Dystrophin Expression in Duchenne Muscular Dystrophy Muscle

(2011) Eric P. Hoffman et al.   AMERICAN JOURNAL OF PATHOLOGY

Impending therapies for Duchenne muscular dystrophy

(2011) Terence A. Partridge   CURRENT OPINION IN NEUROLOGY

Progress in therapy for Duchenne muscular dystrophy

(2011) Rebecca J. Fairclough et al.   EXPERIMENTAL PHYSIOLOGY

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

Current Status of Pharmaceutical and Genetic Therapeutic Approaches to Treat DMD

(2011) Christophe Pichavant et al.   MOLECULAR THERAPY

Pathogenic exon-trapping by SVA retrotransposon and rescue in Fukuyama muscular dystrophy

(2011) Mariko Taniguchi-Ikeda et al.   NATURE

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

Immortalized pathological human myoblasts: towards a universal tool for the study of neuromuscular disorders

(2011) Kamel Mamchaoui et al.   Skeletal Muscle

RNAstructure: software for RNA secondary structure prediction and analysis

(2010) Jessica S Reuter et al.   BMC BIOINFORMATICS

Short Telomeres and Stem Cell Exhaustion Model Duchenne Muscular Dystrophy in mdx/mTR Mice

(2010) Alessandra Sacco et al.   CELL

Exonic Sequences Provide Better Targets for Antisense Oligonucleotides Than Splice Site Sequences in the Modulation of Duchenne Muscular Dystrophy Splicing

(2010) Annemieke Aartsma-Rus et al.   OLIGONUCLEOTIDES

Design of Phosphorodiamidate Morpholino Oligomers (PMOs) for the Induction of Exon Skipping of the Human DMD Gene

(2009) Linda J Popplewell et al.   MOLECULAR THERAPY

Human Splicing Finder: an online bioinformatics tool to predict splicing signals

(2009) François-Olivier Desmet et al.   NUCLEIC ACIDS RESEARCH

Modification of HER2 pre-mRNA alternative splicing and its effects on breast cancer cells

(2008) Jing Wan et al.   INTERNATIONAL JOURNAL OF CANCER

Guidelines for Antisense Oligonucleotide Design and Insight Into Splice-modulating Mechanisms

(2008) Annemieke Aartsma-Rus et al.   MOLECULAR THERAPY

The impact of target site accessibility on the design of effective siRNAs

(2008) Hakim Tafer et al.   NATURE BIOTECHNOLOGY

Dynamics of Co-Transcriptional Pre-mRNA Folding Influences the Induction of Dystrophin Exon Skipping by Antisense Oligonucleotides

(2008) Keng Boon Wee et al.   PLoS One