Annemieke Aartsma-Rus Selected Research

Antisense Oligonucleotides

1/2021	Correction: Pathological mechanism and antisense oligonucleotide-mediated rescue of a non-coding variant suppressing factor 9 RNA biogenesis leading to hemophilia B.
1/2018	A dystrophic Duchenne mouse model for testing human antisense oligonucleotides.
1/2018	A multicenter comparison of quantification methods for antisense oligonucleotide-induced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures.
1/2018	Exon 51 Skipping Quantification by Digital Droplet PCR in del52hDMD/mdx Mice.
10/2016	Antisense Oligonucleotide-mediated Exon Skipping as a Systemic Therapeutic Approach for Recessive Dystrophic Epidermolysis Bullosa.
12/2015	Evaluation of 2'-Deoxy-2'-fluoro Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy.
11/2014	Preclinical studies on intestinal administration of antisense oligonucleotides as a model for oral delivery for treatment of duchenne muscular dystrophy.
7/2014	Antisense-mediated exon skipping: taking advantage of a trick from Mother Nature to treat rare genetic diseases.
2/2014	Peptide conjugation of 2'-O-methyl phosphorothioate antisense oligonucleotides enhances cardiac uptake and exon skipping in mdx mice.
1/2014	Novel Ex Vivo Culture Method for the Study of Dupuytren's Disease: Effects of TGFβ Type 1 Receptor Modulation by Antisense Oligonucleotides.

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Annemieke Aartsma-Rus Research Topics

Disease

73	Duchenne Muscular Dystrophy (Muscular Dystrophy, Becker) 01/2022 - 10/2002
11	Muscular Dystrophies (Muscular Dystrophy) 01/2021 - 12/2007
11	Disease Progression 01/2021 - 01/2013
7	Rare Diseases (Rare Disease) 01/2022 - 01/2013
6	Inborn Genetic Diseases (Disease, Hereditary) 10/2019 - 01/2014
5	Spinal Muscular Atrophy (Progressive Muscular Atrophy) 12/2021 - 12/2015
4	Fibrosis (Cirrhosis) 01/2021 - 04/2014
3	Cardiomyopathies (Cardiomyopathy) 01/2022 - 04/2014
3	Wasting Syndrome (Wasting Disease) 01/2019 - 09/2009
3	Muscle Weakness 01/2016 - 11/2013
2	Inflammation (Inflammations) 01/2021 - 01/2013
2	CADASIL 01/2020 - 01/2019
2	Neuromuscular Diseases (Neuromuscular Disease) 01/2020 - 01/2018
2	Muscular Diseases (Myopathy) 01/2016 - 08/2010
2	Myasthenia Gravis 01/2016 - 04/2014
2	Heart Failure 05/2015 - 04/2014
2	Dilated Cardiomyopathy (Cardiomyopathy, Congestive) 05/2015 - 05/2012
2	Huntington Disease (Huntington's Disease) 02/2014 - 01/2011
1	Sarcoglycanopathies 01/2022
1	Hemophilia B (Haemophilia B) 01/2021
1	Cardiovascular Diseases (Cardiovascular Disease) 01/2021
1	Dementia (Dementias) 01/2020
1	Stroke (Strokes) 01/2020
1	Dysferlinopathy 01/2020
1	Muscular Atrophy (Muscle Atrophy) 01/2017
1	Inclusion Body Myositis 01/2017
1	Myositis (Idiopathic Inflammatory Myopathies) 01/2017
1	Epidermolysis Bullosa Dystrophica (Dystrophic Epidermolysis Bullosa) 10/2016
1	Wounds and Injuries (Trauma) 10/2016
1	Heart Diseases (Heart Disease) 05/2015
1	Bethlem myopathy 04/2014
1	Late Onset Disorders 04/2014
1	Oculopharyngeal Muscular Dystrophy 04/2014
1	Dupuytren Contracture (Dupuytrens Contracture) 01/2014
1	Machado-Joseph Disease (Spinocerebellar Ataxia Type 3) 10/2013
1	Myositis Ossificans (Fibrodysplasia Ossificans Progressiva) 01/2013

Drug/Important Bio-Agent (IBA)

54	DystrophinIBA 01/2022 - 10/2002
27	Proteins (Proteins, Gene)FDA Link 12/2021 - 12/2005
23	Antisense OligonucleotidesIBA 01/2021 - 08/2004
11	Biomarkers (Surrogate Marker)IBA 01/2020 - 04/2014
6	Pharmaceutical PreparationsIBA 01/2022 - 04/2006
6	Messenger RNA (mRNA)IBA 01/2022 - 09/2010
4	RNA (Ribonucleic Acid)IBA 01/2021 - 06/2010
3	MyostatinIBA 01/2022 - 04/2011
3	Nonsense Codon (Nonsense Mutation)IBA 01/2020 - 09/2009
3	Adrenal Cortex Hormones (Corticosteroids)IBA 01/2020 - 01/2013
3	OligonucleotidesIBA 01/2020 - 06/2010
3	polyglutamineIBA 02/2014 - 01/2011
2	Activins (Activin)IBA 01/2022 - 01/2017
2	Simvastatin (Zocor)FDA LinkGeneric 01/2021 - 01/2021
2	nusinersenIBA 01/2020 - 01/2017
2	DysferlinIBA 01/2020 - 08/2010
2	deflazacortIBA 01/2020 - 01/2013
2	Malate Dehydrogenase (Dehydrogenase, Malate)IBA 01/2020 - 07/2014
2	CollagenIBA 01/2020 - 04/2017
2	eteplirsenIBA 01/2019 - 01/2017
2	CytokinesIBA 01/2017 - 01/2013
2	RNA Precursors (Precursor, mRNA)IBA 12/2015 - 07/2014
2	UbiquitinIBA 04/2014 - 11/2013
2	Proteasome Endopeptidase Complex (Proteasome)IBA 04/2014 - 11/2013
2	Ataxin-3IBA 10/2013 - 01/2011
2	PRO051IBA 04/2011 - 12/2007
1	Cytidine DeaminaseIBA 01/2022
1	Biological ProductsIBA 12/2021
1	Factor IX (Coagulation Factor IX)FDA LinkGeneric 01/2021
1	LipidsIBA 01/2021
1	CholesterolIBA 01/2021
1	golodirsenIBA 01/2020
1	Tumor Necrosis Factor Receptors (Tumor Necrosis Factor Receptor)IBA 01/2020
1	Cysteine (L-Cysteine)FDA Link 01/2020
1	Blood Proteins (Serum Proteins)IBA 01/2020
1	Notch3 ReceptorIBA 01/2020
1	Nucleic AcidsIBA 04/2018
1	Methotrexate (Mexate)FDA LinkGeneric 01/2017
1	Interleukin-8 (Interleukin 8)IBA 01/2017
1	Collagen Type VIIIBA 10/2016
1	ProteomeIBA 03/2016
1	AutoantibodiesIBA 01/2016
1	Neurotransmitter Agents (Neurotransmitter)IBA 01/2016
1	Cholinergic ReceptorsIBA 01/2016
1	Dystrophin-Associated ProteinsIBA 06/2015
1	Matrix Metalloproteinase 9 (Gelatinase B)IBA 05/2015
1	ConnectinIBA 05/2015
1	AntibodiesIBA 07/2014
1	Myosin Light Chains (Myosin Essential Light Chain)IBA 07/2014
1	Carbonic Anhydrase IIIIBA 07/2014
1	Electron-Transferring Flavoproteins (Electron Transfer Flavoprotein)IBA 07/2014
1	Alanine (L-Alanine)FDA Link 04/2014
1	Transforming Growth Factors (Transforming Growth Factor)IBA 04/2014
1	Fibronectins (Fibronectin)IBA 04/2014
1	Poly(A)-Binding Protein IIIBA 04/2014
1	Huntingtin ProteinIBA 02/2014
1	Therapeutic UsesIBA 02/2014
1	Peptides (Polypeptides)IBA 02/2014
1	Creatine Kinase (Creatine Phosphokinase)IBA 11/2013
1	Mutant Proteins (Protein, Mutant)IBA 10/2013
1	InterleukinsIBA 01/2013

Therapy/Procedure

24	Therapeutics 01/2022 - 10/2002
2	Intramuscular Injections 04/2014 - 12/2007
1	Precision Medicine 10/2019