Annemieke Aartsma-Rus

Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy.

Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients.

Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense.

Targeted exon skipping in transgenic hDMD mice: A model for direct preclinical screening of human-specific antisense oligonucleotides.

Functional analysis of 114 exon-internal AONs for targeted DMD exon skipping: indication for steric hindrance of SR protein binding sites.

Gene expression profiling to monitor therapeutic and adverse effects of antisense therapies for Duchenne muscular dystrophy.

Exploring the frontiers of therapeutic exon skipping for Duchenne muscular dystrophy by double targeting within one or multiple exons.

Entries in the Leiden Duchenne muscular dystrophy mutation database: an overview of mutation types and paradoxical cases that confirm the reading-frame rule.

Antisense-induced exon skipping for duplications in Duchenne muscular dystrophy.

Antisense-mediated exon skipping: a versatile tool with therapeutic and research applications.

Local dystrophin restoration with antisense oligonucleotide PRO051.

The therapeutic potential of antisense-mediated exon skipping.

Guidelines for antisense oligonucleotide design and insight into splice-modulating mechanisms.

Assessment of the feasibility of exon 45-55 multiexon skipping for Duchenne muscular dystrophy.

In vivo comparison of 2'-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping.

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

Antisense-mediated exon skipping to correct IL-12Rbeta1 deficiency in T cells.

[Experimental therapy in Duchenne muscular dystrophy].

Less is more: therapeutic exon skipping for Duchenne muscular dystrophy.

Development of antisense-mediated exon skipping as a treatment for duchenne muscular dystrophy.

Progress in therapeutic antisense applications for neuromuscular disorders.

Comparative analysis of antisense oligonucleotide sequences targeting exon 53 of the human DMD gene: Implications for future clinical trials.

Therapeutic exon skipping for dysferlinopathies?

A 3 months mild functional test regime does not affect disease parameters in young mdx mice.

Exonic sequences provide better targets for antisense oligonucleotides than splice site sequences in the modulation of Duchenne muscular dystrophy splicing.

Preclinical PK and PD studies on 2'-O-methyl-phosphorothioate RNA antisense oligonucleotides in the mdx mouse model.

Accurate quantification of dystrophin mRNA and exon skipping levels in duchenne muscular dystrophy.

Antisense-mediated modulation of splicing: therapeutic implications for Duchenne muscular dystrophy.

BMP antagonists enhance myogenic differentiation and ameliorate the dystrophic phenotype in a DMD mouse model.

The risks of therapeutic misconception and individual patient (n=1) "trials" in rare diseases such as Duchenne dystrophy.

New insights in gene-derived therapy: the example of Duchenne muscular dystrophy.

Systemic administration of PRO051 in Duchenne's muscular dystrophy.

Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.

Dual exon skipping in myostatin and dystrophin for Duchenne muscular dystrophy.

Opportunities and challenges for the development of antisense treatment in neuromuscular disorders.

Antisense-mediated RNA targeting: versatile and expedient genetic manipulation in the brain.

Targeting several CAG expansion diseases by a single antisense oligonucleotide.

Prednisolone treatment does not interfere with 2'-O-methyl phosphorothioate antisense-mediated exon skipping in Duchenne muscular dystrophy.

Dystrophin quantification and clinical correlations in Becker muscular dystrophy: implications for clinical trials.

Phage display screening without repetitious selection rounds.

Cell-type specific regulation of myostatin signaling.

Assessment of cardiac function in three mouse dystrophinopathies by magnetic resonance imaging.

Comparison of skeletal muscle pathology and motor function of dystrophin and utrophin deficient mouse strains.

The effects of low levels of dystrophin on mouse muscle function and pathology.

Splice modulating therapies for human disease.

Overview on applications of antisense-mediated exon skipping.

Overview on DMD exon skipping.

Overview on AON design.

Antisense oligonucleotide mediated exon skipping as a potential strategy for the treatment of a variety of inflammatory diseases such as rheumatoid arthritis.

Gene therapy for Duchenne muscular dystrophy.

Digenic inheritance of an SMCHD1 mutation and an FSHD-permissive D4Z4 allele causes facioscapulohumeral muscular dystrophy type 2.

The effect of 6-thioguanine on alternative splicing and antisense-mediated exon skipping treatment for duchenne muscular dystrophy.

Antisense-mediated isoform switching of steroid receptor coactivator-1 in the central nucleus of the amygdala of the mouse brain.

Guidance in social and ethical issues related to clinical, diagnostic care and novel therapies for hereditary neuromuscular rare diseases: "translating" the translational.

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

Long-term Exon Skipping Studies With 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides in Dystrophic Mouse Models.

Advances in therapeutic RNA-targeting.

Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases.

Low dystrophin levels increase survival and improve muscle pathology and function in dystrophin/utrophin double-knockout mice.

Innovating therapies for muscle diseases.

Dose-dependent pharmacokinetic profiles of 2'-O-methyl phosphorothioate antisense oligonucleotidesin mdx mice.

Ataxin-3 protein modification as a treatment strategy for spinocerebellar ataxia type 3: removal of the CAG containing exon.

Dystrophin-deficient pigs provide new insights into the hierarchy of physiological derangements of dystrophic muscle.

Exon skipping and gene transfer restore dystrophin expression in hiPSC-cardiomyocytes harbouring DMD mutations.

Exon skipping and gene transfer restore dystrophin expression in human induced pluripotent stem cells-cardiomyocytes harboring DMD mutations.

Antisense-oligonucleotide mediated exon skipping in activin-receptor-like kinase 2: inhibiting the receptor that is overactive in fibrodysplasia ossificans progressiva.

194th ENMC international workshop. 3rd ENMC workshop on exon skipping: towards clinical application of antisense-mediated exon skipping for Duchenne muscular dystrophy 8-10 December 2012, Naarden, The Netherlands.

A 3-base pair deletion, c.9711_9713del, in DMD results in intellectual disability without muscular dystrophy.

DMD transcript imbalance determines dystrophin levels.

Generation of embryonic stem cells and mice for duchenne research.

Autophagy is Impaired in the Tibialis Anterior of Dystrophin Null Mice.

Peptide conjugation of 2'-O-methyl phosphorothioate antisense oligonucleotides enhances cardiac uptake and exon skipping in mdx mice.

Preventing formation of toxic N-terminal huntingtin fragments through antisense oligonucleotide-mediated protein modification.

Novel Ex Vivo Culture Method for the Study of Dupuytren's Disease: Effects of TGFβ Type 1 Receptor Modulation by Antisense Oligonucleotides.

Fibronectin is a serum biomarker for Duchenne muscular dystrophy.

Low dystrophin levels in heart can delay heart failure in mdx mice.

A Novel Feed-Forward Loop between ARIH2 E3-Ligase and PABPN1 Regulates Aging-Associated Muscle Degeneration.

Antisense-mediated exon skipping: Taking advantage of a trick from Mother Nature to treat rare genetic diseases.

Antisense-mediated exon skipping: networking to meet opportunities and to overcome challenges.

The Dynamics of Compound, Transcript, and Protein Effects After Treatment With 2OMePS Antisense Oligonucleotides in mdx Mice.

Development of a web course on gene therapy by the international consortium of gene therapy.