Annemieke M Aartsma-Rus (Annemieke Aarstma-Rus, A Aartsma-Rus, Annemieke Aartsma-Rus)

ORCID

Publication Activity (10 Years)

Years Active: 2018-2024
Publications (10 Years): 56

Top Venues

Nucleic acid therapeutics

Expert opinion on biological therapy

RNA (New York, N.Y.)

PloS one

Leiden University Medical Center

This page only lists publications with an associated author ORCID identifier.

Affiliation History

Leiden University Medical Center Oct 01 2015

Netherlands

Professor of Translational Genetics :: Human Genetics

Publications

Falk Ehmann, Andreas Kuhn, Anna Maria Gerdina Pasmooij, Anthony Humphreys, Arjon Van Hengel, Brian Dooley, Brigitte Anliker, Camilla Svensson, Daniel Capaldi, David Henshall, Emer Cooke, Haiyan Zhou, Hilde Bastaerts, Jeske Smink, Joop Van Gerven, Leonor Enes, Lubomir Nechev, Marcel Hoefnagel, Mariëtte Driessens, Michael Wenger, Oriane Blanquie, Pawel Widomski, Ralf Herold, René Thürmer, Sol Ruiz, Steffen Thirstrup, Susan Goody, Tal Zaks, Valentina Cordoʹ, Annemieke M Aartsma-Rus
Report of the European Medicines Agency Conference on RNA-Based Medicines. Nucleic acid therapeutics 34 (1) (2024)
Falk Ehmann, Andreas Kuhn, Anna Maria Gerdina Pasmooij, Anthony Humphreys, Arjon Van Hengel, Brian Dooley, Brigitte Anliker, Camilla Svensson, Daniel Capaldi, David Henshall, Emer Cooke, Haiyan Zhou, Hilde Bastaerts, Jeske Smink, Joop Van Gerven, Leonor Enes, Lubomir Nechev, Marcel Hoefnagel, Mariëtte Driessens, Michael Wenger, Oriane Blanquie, Pawel Widomski, Ralf Herold, René Thürmer, Sol Ruiz, Steffen Thirstrup, Susan Goody, Tal Zaks, Valentina Cordoʹ, Annemieke M Aartsma-Rus
Report of the European Medicines Agency Conference on RNA-Based Medicines. Nucleic acid therapeutics (2024)
Falk Ehmann, Andreas Kuhn, Anna Maria Gerdina Pasmooij, Anthony Humphreys, Arjon Van Hengel, Brian Dooley, Brigitte Anliker, Camilla Svensson, Daniel Capaldi, David Henshall, Emer Cooke, Haiyan Zhou, Hilde Bastaerts, Jeske Smink, Joop Van Gerven, Leonor Enes, Lubomir Nechev, Marcel Hoefnagel, Mariëtte Driessens, Michael Wenger, Oriane Blanquie, Pawel Widomski, Ralf Herold, René Thürmer, Sol Ruiz, Steffen Thirstrup, Susan Goody, Tal Zaks, Valentina Cordoʹ, Annemieke M Aartsma-Rus
Report of the European Medicines Agency Conference on RNA-Based Medicines. Nucleic acid therapeutics 34 (1) (2024)
Daniel O'Reilly, Willeke van Roon-Mom, Annemieke M Aartsma-Rus, null null
A Guide to Chemical Considerations for the Pre-Clinical Development of Oligonucleotides. Nucleic acid therapeutics (2024)
Falk Ehmann, Andreas Kuhn, Anna Maria Gerdina Pasmooij, Anthony Humphreys, Arjon Van Hengel, Brian Dooley, Brigitte Anliker, Camilla Svensson, Daniel Capaldi, David Henshall, Emer Cooke, Haiyan Zhou, Hilde Bastaerts, Jeske Smink, Joop Van Gerven, Leonor Enes, Lubomir Nechev, Marcel Hoefnagel, Mariëtte Driessens, Michael Wenger, Oriane Blanquie, Pawel Widomski, Ralf Herold, René Thürmer, Sol Ruiz, Steffen Thirstrup, Susan Goody, Tal Zaks, Valentina Cordoʹ, Annemieke M Aartsma-Rus
Report of the European Medicines Agency Conference on RNA-Based Medicines. Nucleic acid therapeutics 34 (1) (2024)
Falk Ehmann, Andreas Kuhn, Anna Maria Gerdina Pasmooij, Anthony Humphreys, Arjon Van Hengel, Brian Dooley, Brigitte Anliker, Camilla Svensson, Daniel Capaldi, David Henshall, Emer Cooke, Haiyan Zhou, Hilde Bastaerts, Jeske Smink, Joop Van Gerven, Leonor Enes, Lubomir Nechev, Marcel Hoefnagel, Mariëtte Driessens, Michael Wenger, Oriane Blanquie, Pawel Widomski, Ralf Herold, René Thürmer, Sol Ruiz, Steffen Thirstrup, Susan Goody, Tal Zaks, Valentina Cordoʹ, Annemieke M Aartsma-Rus
Report of the European Medicines Agency Conference on RNA-Based Medicines. Nucleic acid therapeutics 34 (1) (2024)
Rebecca Schuele, Matthis Synofzik, Holm Graessner, Annemieke M Aartsma-Rus
Advancing ASO therapies from development to implementation. Nature medicine (2024)
Galina Filonova, Annemieke M Aartsma-Rus
Next steps for the optimization of exon therapy for Duchenne muscular dystrophy. Expert opinion on biological therapy 23 (2) (2023)
Willeke M C van Roon-Mom, Chantal Ferguson, Annemieke M Aartsma-Rus
From Failure to Meet the Clinical Endpoint to U.S. Food and Drug Administration Approval: 15th Antisense Oligonucleotide Therapy Approved Qalsody (Tofersen) for Treatment of SOD1 Mutated Amyotrophic Lateral Sclerosis. Nucleic acid therapeutics 33 (4) (2023)
Sarah Engelbeen, Daniel O'Reilly, Davy Van De Vijver, Ingrid E C Verhaart, Maaike van Putten, Vignesh Hariharan, Matthew Hassler, Anastasia Khvorova, Masad J Damha, Annemieke M Aartsma-Rus
Challenges of Assessing Exon 53 Skipping of the Human DMD Transcript with Locked Nucleic Acid-Modified Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy. Nucleic acid therapeutics 33 (6) (2023)
Galina Filonova, Annemieke M Aartsma-Rus
Next steps for the optimization of exon skipping therapy for Duchenne muscular dystrophy. Expert opinion on biological therapy (2023)
Donnie Cameron, Tooba Abbassi-Daloii, Laura G M Heezen, Nienke M van de Velde, Zaïda Koeks, Thom T J Veeger, Melissa T Hooijmans, Salma El Abdellaoui, Sjoerd G van Duinen, Jan J G M Verschuuren, Maaike van Putten, Annemieke M Aartsma-Rus, Vered Raz, Pietro Spitali, Erik H Niks, Hermien E Kan
Diffusion-tensor magnetic resonance imaging captures increased skeletal muscle fibre diameters in Becker muscular dystrophy. Journal of cachexia, sarcopenia and muscle (2023)
Galina Filonova, Annemieke M Aartsma-Rus
Next steps for the optimization of exon therapy for Duchenne muscular dystrophy. Expert opinion on biological therapy 23 (2) (2023)
Judith van Deutekom, Chantal Beekman, Suzanne Bijl, Sieto Bosgra, Rani van den Eijnde, Dennis Franken, Bas Groenendaal, Bouchra Harquouli, Anneke Janson, Paul Koevoets, Melissa Mulder, Daan Muilwijk, Galyna Peterburgska, Bianca Querido, Janwillem Testerink, Ruurd Verheul, Peter de Visser, Rudie Weij, Annemieke M Aartsma-Rus, Jukka Puoliväli, Timo Bragge, Charles O'Neill, Nicole A Datson
Next Generation Exon 51 Skipping Antisense Oligonucleotides for Duchenne Muscular Dystrophy. Nucleic acid therapeutics (2023)
Mathilde Doisy, Ophélie Vacca, Claire Fergus, Talia Gileadi, Minou Verhaeg, Amel Saoudi, Thomas Tensorer, Luis Garcia, Vincent P Kelly, Federica Montanaro, Jennifer E Morgan, Maaike van Putten, Annemieke M Aartsma-Rus, Cyrille Vaillend, Francesco Muntoni, Aurelie T Goyenvalle
Networking to Optimize Dmd exon 53 Skipping in the Brain of mdx52 Mouse Model. Biomedicines 11 (12) (2023)
Mathilde Doisy, Ophélie Vacca, Claire Fergus, Talia Gileadi, Minou Verhaeg, Amel Saoudi, Thomas Tensorer, Luis Garcia, Vincent P Kelly, Federica Montanaro, Jennifer E Morgan, Maaike van Putten, Annemieke M Aartsma-Rus, Cyrille Vaillend, Francesco Muntoni, Aurelie T Goyenvalle
Networking to Optimize Dmd exon 53 Skipping in the Brain of mdx52 Mouse Model. Biomedicines 11 (12) (2023)
Annemieke M Aartsma-Rus, Willeke M C van Roon-Mom, Marlen C Lauffer, Christine Siezen, Britt Duijndam, Tineke Coenen-de Roo, Rebecca Schüle, Matthis Synofzik, Holm Graessner
Development of tailored splice switching oligonucleotides for progressive brain disorders in Europe: development, regulation and implementation considerations. RNA (New York, N.Y.) (2023)
Laura G M Heezen, Tamim Abdelaal, M van Putten, Annemieke M Aartsma-Rus, Ahmed Mahfouz, Pietro Spitali
Spatial transcriptomics reveal markers of histopathological changes in Duchenne muscular dystrophy mouse models. Nature communications 14 (1) (2023)
Mirko Signorelli, Roula Tsonaka, Annemieke M Aartsma-Rus, Pietro Spitali
Multiomic characterization of disease progression in mice lacking dystrophin. PloS one 18 (3) (2023)
Annemieke M Aartsma-Rus, Willeke M C van Roon-Mom, Marlen C Lauffer, Christine Siezen, Britt Duijndam, Tineke Coenen-de Roo, Rebecca Schüle, Matthis Synofzik, Holm Graessner
Development of tailored splice-switching oligonucleotides for progressive brain disorders in Europe: development, regulation, and implementation considerations. RNA (New York, N.Y.) 29 (4) (2023)
Mirko Signorelli, Roula Tsonaka, Annemieke M Aartsma-Rus, Pietro Spitali
Multiomic characterization of disease progression in mice lacking dystrophin. PloS one 18 (3) (2023)
Remko Goossens, Nisha Verwey, Yavuz Ariyurek, Fred Schnell, Annemieke M Aartsma-Rus
DMD antisense oligonucleotide mediated exon skipping efficiency correlates with flanking intron retention time and target position within the exon. RNA biology 20 (1) (2023)
Galina Filonova, Annemieke M Aartsma-Rus
Next steps for the optimization of exon therapy for Duchenne muscular dystrophy. Expert opinion on biological therapy (2023)
Mirko Signorelli, Roula Tsonaka, Annemieke M Aartsma-Rus, Pietro Spitali
Multiomic characterization of disease progression in mice lacking dystrophin. PloS one 18 (3) (2023)
Annemieke M Aartsma-Rus, Willeke M C van Roon-Mom, Marlen C Lauffer, Christine Siezen, Britt Duijndam, Tineke Coenen-de Roo, Rebecca Schüle, Matthis Synofzik, Holm Graessner
Development of tailored splice-switching oligonucleotides for progressive brain disorders in Europe: development, regulation, and implementation considerations. RNA (New York, N.Y.) 29 (4) (2023)
Galina Filonova, Annemieke M Aartsma-Rus
Next steps for the optimization of exon therapy for Duchenne muscular dystrophy. Expert opinion on biological therapy 23 (2) (2023)
Annemieke M Aartsma-Rus, Willeke M C van Roon-Mom, Marlen C Lauffer, Christine Siezen, Britt Duijndam, Tineke Coenen-de Roo, Rebecca Schüle, Matthis Synofzik, Holm Graessner
Development of tailored splice-switching oligonucleotides for progressive brain disorders in Europe: development, regulation, and implementation considerations. RNA (New York, N.Y.) 29 (4) (2023)
Annemieke M Aartsma-Rus, Willeke M C van Roon-Mom, Marlen C Lauffer, Christine Siezen, Britt Duijndam, Tineke Coenen-de Roo, Rebecca Schüle, Matthis Synofzik, Holm Graessner
Development of tailored splice-switching oligonucleotides for progressive brain disorders in Europe: development, regulation, and implementation considerations. RNA (New York, N.Y.) 29 (4) (2023)
Annemieke M Aartsma-Rus, Alejandro Garanto, Willeke M C van Roon-Mom, Erin M McConnell, Victoria Suslovitch, Winston X Yan, Jonathan K Watts, Timothy W Yu
Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1 Exon Skipping Antisense Oligonucleotides. Nucleic acid therapeutics 33 (1) (2022)
Annemieke M Aartsma-Rus, Alejandro Garanto, Willeke M C van Roon-Mom, Erin M McConnell, Victoria Suslovitch, Winston X Yan, Jonathan K Watts, Timothy W Yu
Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1 Exon Skipping Antisense Oligonucleotides. Nucleic acid therapeutics 33 (1) (2022)
Annemieke M Aartsma-Rus, Alejandro Garanto, Willeke M C van Roon-Mom, Erin M McConnell, Victoria Suslovitch, Winston X Yan, Jonathan K Watts, Timothy W Yu
Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1 Exon Skipping Antisense Oligonucleotides. Nucleic acid therapeutics 33 (1) (2022)
Damien Evéquoz, Ingrid E C Verhaart, Davy van de Vijver, Wolfgang Renner, Annemieke M Aartsma-Rus, Christian J Leumann
7',5'-alpha-bicyclo-DNA: new chemistry for oligonucleotide exon splicing modulation therapy. Nucleic acids research 49 (21) (2022)
Yu C J Chey, Jayshen Arudkumar, Annemieke M Aartsma-Rus, Fatwa Adikusuma, Paul Q Thomas
CRISPR applications for Duchenne muscular dystrophy: From animal models to potential therapies. WIREs mechanisms of disease (2022)
Annemieke M Aartsma-Rus, Alejandro Garanto, Willeke M C van Roon-Mom, Erin M McConnell, Victoria Suslovitch, Winston X Yan, Jonathan K Watts, Timothy W Yu
Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1 Exon Skipping Antisense Oligonucleotides. Nucleic acid therapeutics (2022)
Annemieke M Aartsma-Rus, Alejandro Garanto, Willeke M C van Roon-Mom, Erin M McConnell, Victoria Suslovitch, Winston X Yan, Jonathan K Watts, Timothy W Yu
Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1 Exon Skipping Antisense Oligonucleotides. Nucleic acid therapeutics 33 (1) (2022)
Annemieke M Aartsma-Rus, Alejandro Garanto, Willeke M C van Roon-Mom, Erin M McConnell, Victoria Suslovitch, Winston X Yan, Jonathan K Watts, Timothy W Yu
Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1 Exon Skipping Antisense Oligonucleotides. Nucleic acid therapeutics 33 (1) (2022)
Annemieke M Aartsma-Rus, Alejandro Garanto, Willeke M C van Roon-Mom, Erin M McConnell, Victoria Suslovitch, Winston X Yan, Jonathan K Watts, Timothy W Yu
Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1 Exon Skipping Antisense Oligonucleotides. Nucleic acid therapeutics 33 (1) (2022)
Dongsheng Duan, Kevin M Flanigan, Annemieke M Aartsma-Rus
Letter by Duan et al Regarding Article, "Therapeutic Exon Skipping Through a CRISPR-Guided Cytidine Deaminase Rescues Dystrophic Cardiomyopathy In Vivo". Circulation 145 (18) (2022)
Sarah Engelbeen, Svetlana Pasteuning-Vuhman, Joke Boertje-van der Meulen, Rubina Parmar, Klaus Charisse, Laura Sepp-Lorenzino, Muthiah Manoharan, Annemieke M Aartsma-Rus, Maaike van Putten
Efficient Downregulation of Alk4 in Skeletal Muscle After Systemic Treatment with Conjugated siRNAs in a Mouse Model for Duchenne Muscular Dystrophy. Nucleic acid therapeutics (2022)
Suzan M Hammond, Annemieke M Aartsma-Rus, Sandra Alves, Sven Even F Borgos, Ronald A M Buijsen, Rob W J Collin, Giuseppina Covello, Michela Alessandra Denti, Lourdes Ruiz Desviat, Lucía Echevarría, Camilla Foged, Gisela Gaina, Alejandro Garanto, Aurelie T Goyenvalle, Magdalena Guzowska, Irina Kholodnyuk, David R Jones, Sabine Krause, Taavi Lehto, Marisol Montolio, Willeke M C van Roon-Mom, Virginia Arechavala-Gomeza
Delivery of oligonucleotide-based therapeutics: challenges and opportunities. EMBO molecular medicine 13 (4) (2021)
Dongsheng Duan, Nathalie Goemans, Shin'ichi Takeda, Eugenio Mercuri, Annemieke M Aartsma-Rus
Duchenne muscular dystrophy. Nature reviews. Disease primers 7 (1) (2021)
Annemieke M Aartsma-Rus
'N of 1' therapies need a better model. Nature medicine 27 (6) (2021)
Matthis Synofzik, Willeke M C van Roon-Mom, Georg Marckmann, Hermine A van Duyvenvoorde, Holm Graessner, Rebecca Schüle, Annemieke M Aartsma-Rus
Preparing n-of-1 Antisense Oligonucleotide Treatments for Rare Neurological Diseases in Europe: Genetic, Regulatory, and Ethical Perspectives. Nucleic acid therapeutics (2021)
Annemieke M Aartsma-Rus, Elizabeth Vroom, Daniel O'Reilly
The Role of Patient Involvement When Developing Therapies. Nucleic acid therapeutics (2021)
Elsa C Kuijper, Atze J Bergsma, W W M Pim Pijnappel, Annemieke M Aartsma-Rus
Opportunities and challenges for antisense oligonucleotide therapies. Journal of inherited metabolic disease 44 (1) (2020)
Pin Lyu, Kyung Whan Yoo, Manish Kumar Yadav, Anthony Atala, Annemieke M Aartsma-Rus, Maaike van Putten, Dongsheng Duan, Manish Kumar Yadav
Sensitive and reliable evaluation of single-cut sgRNAs to restore dystrophin by a GFP-reporter assay. PloS one 15 (9) (2020)
Simon A Krooss, Sonja Werwitzke, Johannes Kopp, Alice Rovai, Dirk Varnholt, Amelie S Wachs, Aurelie T Goyenvalle, Annemieke M Aartsma-Rus, Michael Ott, Andreas Tiede, Jörg Langemeier, Jens Bohne
Pathological mechanism and antisense oligonucleotide-mediated rescue of a non-coding variant suppressing factor 9 RNA biogenesis leading to hemophilia B. PLoS genetics 16 (4) (2020)
Pietro Spitali, Irina Zaharieva, Stefan Bohringer, Monika Hiller, Amina Chaouch, Andreas Roos, Chiara Scotton, Mireille Claustres, Luca Bello, Craig M McDonald, Eric P Hoffman, null null, Zaida Koeks, Eka H E D Suchiman, Sebahattin Cirak, Mariacristina Scoto, Mojgan Reza, Peter A C 't Hoen, Erik H Niks, Sylvie Tuffery-Giraud, Hanns Lochmüller, Alessandra Ferlini, Francesco Muntoni, Annemieke M Aartsma-Rus
TCTEX1D1 is a genetic modifier of disease progression in Duchenne muscular dystrophy. European journal of human genetics : EJHG 28 (6) (2020)
Anne-Fleur E Schneider, Annemieke M Aartsma-Rus
Developments in reading frame restoring therapy approaches for Duchenne muscular dystrophy. Expert opinion on biological therapy 21 (3) (2020)
Ingrid E C Verhaart, Annemieke M Aartsma-Rus
Therapeutic developments for Duchenne muscular dystrophy. Nature reviews. Neurology 15 (7) (2020)
Maria Inês Alvelos, Mirko Brüggemann, Fx Reymond Sutandy, Jonàs Juan-Mateu, Maikel Luis Colli, Anke Busch, Miguel Lopes, Ângela Castela, Annemieke M Aartsma-Rus, Julian König, Kathi Zarnack, Decio L Eizirik
The RNA-binding profile of the splicing factor SRSF6 in immortalized human pancreatic β-cells. Life science alliance 4 (3) (2020)
Gido Gravesteijn, Leon P Munting, Maurice Overzier, Adriaan A Mulder, Ingrid Hegeman, Marc P P Derieppe, Abraham J Koster, Sjoerd G van Duinen, Onno C Meijer, Annemieke M Aartsma-Rus, Louise van der Weerd, Carolina R Jost, Arn M J M van den Maagdenberg, Julie W Rutten, Saskia A J Lesnik Oberstein
Progression and Classification of Granular Osmiophilic Material (GOM) Deposits in Functionally Characterized Human NOTCH3 Transgenic Mice. Translational stroke research 11 (3) (2019)
Annemieke M Aartsma-Rus, Johan T den Dunnen
Phenotype predictions for exon deletions/duplications: A user guide for professionals and clinicians using Becker and Duchenne muscular dystrophy as examples. Human mutation 40 (10) (2019)
Annemieke M Aartsma-Rus, Maaike van Putten
The use of genetically humanized animal models for personalized medicine approaches. Disease models & mechanisms 13 (2) (2019)
Mirko Signorelli, Burcu Ayoglu, Camilla Johansson, Hanns Lochmüller, Volker Straub, Francesco Muntoni, Erik H Niks, Roula Tsonaka, Anja Persson, Annemieke M Aartsma-Rus, Peter Nilsson, Cristina Al-Khalili Szigyarto, Pietro Spitali
Longitudinal serum biomarker screening identifies malate dehydrogenase 2 as candidate prognostic biomarker for Duchenne muscular dystrophy. Journal of cachexia, sarcopenia and muscle 11 (2) (2019)
Hanns Lochmüller, Dorota M Badowska, Rachel Thompson, Nine V Knoers, Annemieke M Aartsma-Rus, Ivo Glynne Gut, Libby Wood, Tina Harmuth, Andre Durudas, Holm Graessner, Franz Schaefer, Olaf Riess, null null, null null, null null
RD-Connect, NeurOmics and EURenOmics: collaborative European initiative for rare diseases. European journal of human genetics : EJHG 26 (6) (2018)

Annemieke M Aartsma-Rus.css-eqlu8i{font-size:small;padding-left:4px;} (Annemieke Aarstma-Rus, A Aartsma-Rus, Annemieke Aartsma-Rus)

Publication Activity (10 Years)

Top Topics

Top Venues

Annemieke M Aartsma-Rus (Annemieke Aarstma-Rus, A Aartsma-Rus, Annemieke Aartsma-Rus)