Dissemination
DCRT's development of antisense oligonucleotide (ASO) treatment for individual patients (N-of-1) with genetic brain or eye disorders are addressed in numerous talks and poster presentations at (inter)national meetings and conferences.
DCRT's goals and challenges are outlined in the 2021 publication of Annemieke Aartsma-Rus in Nature Medicine. Together with the N=1 Collaborative, the DCRT published consensus guidelines for the development of individualized ASOs on a global level, and, in collaboration with the 1Mutation1Medicine (1M1M) consortium, we wrote an article about our plan for the development of treatments for individual patients in Europe.
Talks
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Cold Spring Harbor NAT meeting
22 March 2025
On Saturday, 22 March, Annemieke Aartsma-Rus will give a presentation at the 9th Cold Spring Harbor Laboratory meeting on Nucleic Acid Therapies.
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Annual Clinical Genetics meeting 2025
21 March 2025
Marlen Lauffer, senior researcher at the DCRT, will present on our work on individualized ASOs at the 2025 Annual Clinical Genetics meeting in Los Angeles, USA. Marlen is co-author of N1C's variant selection guidelines and member of the N1C patient identification working group.
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Swiss RNA Therapeutics Summit
23 January 2025
Annemieke Aartsma-Rus gave a presentation entitled "From Duchenne exon skipping to N=1 treatment: Applying lessons learned for (very) rare disease patients" at the Swiss RNA Therapeutics Summit in Bern, Switzerland. She highlighted the work of the DCRT and stressed the importance of collaboration and sharing. DCRT’s expert advice was also mentioned in a talk presenting work from Algara Goga and Johannes Häberle, who are trying to develop ASOs for a Swiss N=1 patient with a metabolic disorder.
Annemieke was also interviewed by Swiss Radio to talk about N=1 therapies and her work in Duchenne.
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N1C Variant Workshop
10 December 2024
DCRT's Marlen Lauffer and David Cheerie (The Hospital for Sick Children, Toronto) co-authored the N1C variant selection guidelines. Today, N1C organized a variant workshop where Marlen and David presented the guidelines and answered questions from the participants.
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DCRT at the Oligonucleotide Therapeutics Symposium in Montreal
6-9 October 2024
The annual meeting of the Oligonucleotide Therapeutics Society will take place in Montreal, Canada. Many people from the DCRT will present. Come and find us at the poster sessions!
Annemieke Aartsma-Rus has been invited as keynote speaker! On Sunday, October 6th, she will give a presentation entitled
"From rare to extremely rare: Applying lessons learned from Duchenne muscular dystrophy antisense oligonucleotide mediated exon skipping to developing individualized treatment".
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Inaugural Annual Meeting of the N=1 Collaborative
5 October 2024
The first N=1 Collaborative annual meeting, entitled "Individual Journeys, Shared Discoveries", was held in Montreal, Canada, on October 5th. Willeke van Roon-Mom, Marlen Lauffer, Annemieke Aartsma-Rus and Jack Morgan presented on behalf of the DCRT.
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RNA Therapeutics Symposium: Unlocking the RNA’s Potential in Medicine
25-27 September 2024
Annemieke Aartsma-Rus presented on the DCRT at the RNA Therapeutics Symposium in Porto, Portugal.
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Summerschool on genetic therapies in Poland
From 3rd to 13th September 2024, Marlen Lauffer was teaching at a Summer School in Wroclaw (Poland). The course focused on genetic therapies, covering different types of strategies, also discussing the work of the DCRT.
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3rd Dutch Antisense Therapeutics Symposium
23-24 May 2024
Many DCRT researchers participated in the 3rd Dutch Antisense Therapeutics Symposium which was held in Rotterdam on 23 and 24 May 2024.
DCRT's Prof. Rob Collin was invited as one of the keynote speakers. The DCRT session featured presentations by Prof. Annemieke Aartsma-Rus, Prof. Matthis Synofzik, Prof. Bart van de Warrenburg and Floor Nieuwenhuijse.
There were poster presentations from Redmar van den Berg, Nick van Dullemen, Janina Lösch, Iris Ensink, Marlen Lauffer, Anouk Spruit and Jack Morgan (who won the award for best poster).
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EuroNDD 2024 - ERN ITHACA
4-5 April 2024
Marlen Lauffer will present DCRT's work on individualized antisense oligonucleotide therapies for patients with rare neurological disorders at the EuroNDD meeting in Lisbon. This 2-day workshop focuses on the multifaceted care and research of rare genetic neurodevelopmental disorders (NDD).
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37th annual APS conference in Kassel
15 March 2024
DCRT's Marlen Lauffer will give an invited talk on Genetic therapies for neurometabolic disorders at the APS (Association for pediatric metabolic disorders) in Kassel, Germany.
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N1C panel discussion with DCRT's Marlen Lauffer
4 March 2024
On the 4th of March, 2024, Dr. Marlen Lauffer, Dr. Lynn Bush and Margaret Meserve will organize a panel discussion about 'Connecting, Collaborating, Communicating, Challenges: Sharing experiences navigating family conversations in an n-of-1 investigational treatment setting'. The N=1 Collaborative is hosting the panel session on its website. Please register on the site if you want to attend (link below).
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Zurich University Seminar series
26 February 2024
DCRT's Annemieke Aartsma-Rus gave a presentation on antisense oligonucleotides in n-of-1 trials at the Zurich University seminar series.
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Italian Society of Pharmacology meeting
23 February 2024
Annemieke Aartsma-Rus gave a presentation entitled "Antisense oligonucleotides in rare diseases: lessons learned from neuromuscular disorders to N-of-1 treatment in brain diseases" at the rare disease meeting of the Italian Society of Pharmacology in Bari.
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Gene and RNA Therapy Center Tuebingen webinar
12 February 2024
Annemieke Aartsma-Rus gave a webinar for the Gene and RNA Therapy Center in Tübingen, one of the partner institutes in the 1M1M consortium.
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Webinar by Annemieke Aartsma-Rus
12 February 2024
Annemieke Aartsma-Rus gave a webinar about DCRT's work for the Gene and RNA Therapy Center in Tübingen.
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48th annual meeting of the society for neuropediatrics
17 November 2023
DCRT's senior researcher Marlen Lauffer presented her poster entitled 'Individualized antisense oligonucleotide therapies for patients with rare neurological disorders' at the 48th annual meeting of the society for neuropediatrics (GNP) from 16th to 18th November 2023 in Dortmund.
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TIDES Europe conference in Amsterdam
31 October - 1 November 2023
DCRT's Willeke van Roon-Mom and Rob Collin will present at the TIDES 2023 conference in Amsterdam on 31 October and 1 November 2023.
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DCRT presented at the 19th annual meeting of the Oligonucleotide Therapeutics Society
26 October 2023
The DCRT presented at the 19th annual meeting of the OTS in Barcelona. There were poster presentations from Anouk, Marlen, Bianca and Emilio.
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Marlen Lauffer N1C seminar series
18 September 2023
Marlen Lauffer gave a presentation in the N1C seminar series entiteld 'Selecting the right candidate: which disease-causing variants are eligible for N=1 ASOs?'. Her presentation was recorded and is available on the N1C website.
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Rob Collin presented at the 23rd annual EuRetina Congress
6 October 2023
Rob Collin presented his research on antisense oligonucleotides at the 23rd annual EuRetina congress in Amsterdam.
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2023 SSIEM Annual Symposium, Jerusalem
1 September 2023
On September 1st Annemieke Aartsma-Rus presented on the DCRT, 1M1M and the N-of-1 collaborative at the SSIEM2023 meeting in Jerusalem. She outlined how lessons from exon skipping therapy development for Duchenne can be applied to other diseases. She also stressed that N-of-1 treatment can only be realized by collaborations between different stakeholders from around the world. (photo courtesy of Anne Bechet)
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Annemieke Aartsma-Rus visited the Sheba Medical Center in Israel
28 August 2023
Annemieke Aartsma-Rus visited the Sheba Medical Center in Israel to give a lecture on Duchenne and to discuss N-of-1 therapy development with collaborators Nofar Mor and Sharon Avkim on August 28th. Discussions were very productive with mutual learning and recognition of the difficult path to make N-of-1 treatment with antisense oligonucleotides a reality.
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Cold Spring Harbor Asia Conference
19 - 22 June 2023
DCRT's Willeke van Roon-Mom was invited to the CSH Asia conference in Ajawi, Japan, from 19-22 June 2023. She gave a presentation entiteld 'Splice modulating RNA targeting therapies for brain disorders: from treating many to treating one'.
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Cure CLCN4 conference in London
16 and 17 June 2023
The DCRT was well represented at the conference of the Cure CLCN4 charity in London last week. Bianca Zardetto presented her work on our variant screening tool in a poster and Marlen Lauffer gave a talk on DCRT's RNA therapy for ultra rare disorders.
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ESHG conference 2023
12 June 2023
Marlen Lauffer represented the DCRT at the 2023 conference of the European Society of Human Genetics in Glasgow. She presented a poster entitled Patient and variant stratification for personalized genetic treatments of nano-rare diseases.
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Cold Spring Harbor Laboratory meeting on Nucleic Acid Therapies
24 March 2023
DCRT's Willeke van Roon-Mom (on the left) gave an invited talk entitled 'The ups-and-downs of preclinical development of splice modulating RNA targeting therapies for neurological disorders' at the Cold Spring Harbor meeting on nucleic acid therapies in Cold Spring Harbor, NY.
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34th Annual Meeting of the German Society of Human Genetics
15 - 17 March 2023
Last Friday, DCRT's Ype Elgersma gave an invited talk at the 34th annual meeting of the GfH in Kassel, Germany, entitled 'New insights and ASO treatment developments for Angelman Syndrome'. Marlen Lauffer also presented at this meeting on DCRT's variant selection for individualized genetic therapies for ultra-rare diseases.
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N=1 Collaborative seminar series
13 February 2023
Today, Annemieke Aartsma-Rus gave a talk in the N1C seminar series. She presented on a recently published paper in Nucleic Acid Therapeutics on the N1C consensus guidelines for N=1 exon skipping design and in vitro development.
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X-talk: Visions for a new era of genetic medicines in neurological disorders
3 February 2023
Willeke van Roon-Mom was invited at the Genetic Risks X-talk at UMC Utrecht. She presented DCRT's advances in the field of n=1 therapeutics for genetic neurological disorders.
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Rare-Med symposium
8 December 2022
DCRT's Willeke van Roon-Mom presented at the symposium of the RARE-MED consortium in Ghent on 8 December.
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MEDVIA Science for Health conference
21 November 2022
Willeke van Roon-Mom gave a talk at the MEDVIA Science for Health conference: Advancing Gene Therapy in Brussels.
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Symposium on the development of orphan drugs
17 November 2022
Willeke van Roon-Mom participated in a panel discussion at a symposium on the development of orphan drugs organized by the association for innovative medicine in the Netherlands.
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Meeting of the German society for rare neurological diseases
10 November 2022
Annemieke Aartsma-Rus presented on DCRT at the meeting of the German society for rare neurological diseases.
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Translational neuroscience symposium
2 November 2022
The Neuroscience theme of Leiden University Medical Center organized a translational neuroscience symposium on November 2nd In Leiden. Marlen, Anouk, Bianca and Laurie each presented a poster on their research at DCRT.
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Symposium about DNA for the general public
30 October 2022
Annemieke Aartsma-Rus gave a talk on RNA therapy and the DCRT at a symposium about DNA for the general public in the botanical gardens in Leiden. Nice initiative from MGC.
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Biotech Thursday: Going for the Cure
On 20 October 2022, Willeke van Roon-Mom presented on DCRT at 'Biotech Thursday: Going for the Cure' organized by HollandBIO.
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18th OTS meeting
2-5 October 2022
The DCRT attended a very successful and well-organized 18th annual OTS meeting in Phoenix, Arizona. We even received a few poster awards!
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European Society for Cataract and Refractive Surgery Annual Meeting
17 September 2022
Professor Rob Collin presented his work and the activities of the DCRT at the European Society for Cataract and Refractive Surgery Annual Meeting in Milan, Italy.
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44th Congress of the SEBBM in Malaga
6 September 2022
Annemieke Aartsma-Rus, co-founder of the DCRT, will give a presentation at the SEBBM congress entitled 'RNA-based therapies for rare diseases treatment'. If you would like to find out more about DCRT's activites, you are welcome to come and talk with Annemieke after her plenary lecture.
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FEBS special session in Lisbon
12 July 2022
In the FEBS Special Session Annemieke Aartsma-Rus gave a presentation entitled: the Dutch Center of RNA Therapeutics: developing mutation-specific antisense oligonucleotide therapies for patients with eye and brain diseases carrying unique mutations.
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SIBBM in Rome
21-22 June 2022
On 21 and 22 June 2022, the Italian Society of Biophysics and Molecular Biology organized an RNA conference in Rome. Willeke van Roon-Mom, one of the invited speakers, presented on DCRT's RNA therapy.
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NVGCT symposium
15-16 June 2022
Rob Collin represented DCRT at the annual symposium of the Netherlands Society of Gene and Cell Therapy. He gave a talk about the DCRT for o.a. patient representatives.
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ESHG 2022
11-14 June 2022
At the 2022 conference of the European Society of Human Genetics in Vienna, Marlen Lauffer presented a poster on DCRT's work.
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Muscles2meet
10-11 June 2022
Annemieke Aartsma-Rus was the keynote speaker at the muscles2meet symposium, an initiative of the Prinses Beatrix spierfonds for PhD students working on neuromuscular diseases.
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1st Dutch Antisense Therapeutics Symposium
3 June 2022
LUMC, Radboudumc and Erasmus MC represented DCRT with talks and poster presentations at the 1st Dutch Antisense Therapeutics Symposium.
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63rd Annual meeting of the Japanese Society of Neurology
18-21 May 2022
DCRT's Annemieke Aartsma-Rus gave a presentation entitled: "DCRT: a center to develop antisense oligonucleotide therapies for patients with ultrarare mutations".
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2022 FDA/DIA Oligonucleotide-based Therapeutics Conference
26 April 2022
Annemieke Aartsma-Rus presented at the DIA FDA meeting on oligonucleotides during the session on Individualized Antisense Oligonucleotide Products.
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Centre for DNA Fingerprinting and Diagnostics (CDFD) one-day symposium
27 January 2022
11:00 AM CET
Professor Dr. Annemieke Aartsma Rus
Video 6:24:17
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Festival of Genomics & Biodata
25 January 2022
11:30 GMT / 12:30 CET / 06:30 EST
Professor Annemieke Aartsma-Rus
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Oligonucleotide Therapeutics SocietyLijstitem 1
21 November 2021
8:00 PST/17:00 CET/11:00 EST
Professor Willeke van Roon-Mom
Posters 2025
3-4 April, ISSCR symposium Athens, Greece. Neurotoxicity platform development of human iPSC-derived neuronal cultures. Anouk Spruit et al.
Posters 2024
6-9 October, OTS 2024, Montreal, Canada. Personalized antisense oligonucleotide screening for a Stargardt disease patient carrying the deep-intronic c.859-506G>C
ABCA4 variant using human
in vitro models.
Edwin van Oosten et al.
6-9 October, OTS 2024, Montreal, Canada. Versatile in vitro toolbox for disease modeling, ASO-screening and neurotoxicity studies. Marlen Lauffer et al.
6-9 October, OTS 2024, Montreal, Canada. In Silico-Guided Selection of Target Sequences for RNase H1-Dependent Antisense Oligonucleotides.
Emilio Harris-Mostert et al.
6-9 October, OTS 2024, Montreal, Canada. Development and Validation of a Rodent Model Protocol Sensitive to Antisense Oligonucleotides Neurotoxic Effects. Raul Andres Santamaria et al.
6-9 October, OTS 2024, Montreal, Canada. Antisense oligonucleotide-mediated exon skipping treatments for neurodevelopmental disorders associated with tandem-repeat domain missense variants in ANKRD17. Jack Morgan et al.
11-13 March, Stem Cells in Neuroscience, Tübingen, Germany. Generation of isogenic hiPSC lines to elucidate the pathomechanism of the recurrent c.1027G>A, p.Gly343Arg variant in ACTL6B. Bianca Zardetto et al.
4-5 April, EuroNDD, Lisbon, Portugal. Individualized antisense oligonucleotide therapies for patients with rare neurological disorders. Marlen Lauffer et al.
23-24 May, DATS, Rotterdam, NL. Recommendations for the identification of genetic variants eligible for individualized antisense oligonucleotide treatments.
Marlen Lauffer et al.
23-24 May, DATS, Rotterdam, NL. Development of an individualized splice-modulating antisense oligonucleotide therapy for patients suffering from NDUFAF6-associated Leigh Syndrome. Iris Ensink et al.
23-24 May, DATS, Rotterdam, NL. ExonViz: an application to visualize transcripts and variants. Redmar van den Berg et al.
23-24 May, DATS, Rotterdam, NL. Platform to screen for ASO-mediated neurotoxicity in human control-derived iPSC neuronal cultures. Anouk Spruit et al.
23-24 May, DATS, Rotterdam, NL. Using a midi-gene assay for the development of an allele-specific gapmer antisense oligonucleotide targeting a heterozygous ACTL6B variant. Nick van Dullemen et al.
23-24 May, DATS, Rotterdam, NL. Evaluation of gapmer ASO candidates targeting a heterozygous ACTL6B variant in neurons transdifferentiated from patient fibroblasts. Janina Lösch et al.
1-4 June, ESHG, Berlin, Germany. ExonViz: an application to generate and visualize transcripts including variants. Redmar van den Berg et al.
Posters 2023
Human in vitro toolbox for ASO screening and neurotoxicity studies. Marlen Lauffer et al.
Patient and variant stratification for personalized genetic treatments of nano-rare diseases. Marlen Lauffer et al.
Exploring the 5'UTR-mediated regulation of ataxin-1 protein expression using splice-modulating antisense oligonucleotides. Laurie Kerkhof et al.
Towards allele-selective gapmer ASO design. Emilio Harris-Mostert et al.
Development of a customized antisense oligonucleotide-based therapeutic strategy for Stargardt disease. Edwin van Oosten et al.
Transdifferentiation of fibroblasts to oligodendrocytes as a platform for ASO-screening. Dion den Hertog et al.
Splice-modulating antisense oligonucleotide treatments for PLP1-associated HEMS. Bianca Zardetto et al.
Rational design and testing of allele-selective gapmer antisense oligonucleotides. Emilio Harris-Mostert et al.
A retrospective analysis of the parameter guidelines for AON design on exon skip efficacy in ATXN1 and ATXN3. Frederique Rotteveel et al.
Posters 2022
Patient and variant stratification for personalized antisense oligonucleotide treatments. Marlen C. Lauffer et al.
Personalized antisense-oligonucleotide therapy for a patient with beta-propeller protein-associated neurodegeneration. Anouk Spruit et al.
RNA therapy development for SCA1 using RNA degrading- and splice-modulating antisense oligonucleotides. Laurie Kerkhof et al.
The Dutch Center for RNA Therapeutics: a center to develop antisense oligonucleotide therapies for patients with nano-rare mutations. Marlen Lauffer et al.
ExonCheck: a tool to prioritize ultra-rare disease patients for personalized antisense oligonucleotide therapies. Iris Huitink et al.
Development of an allele-specific antisense oligonucleotide targeting a heterozygous ACTL6B variant. Max Voslamber et al.
Personalized antisense oligonucleotide therapies for rare neurodevelopmental disorders. Bianca Zardetto et al.
PUBLICATIONS
Consensus guidelines for assessing eligibility of pathogenic DNA variants for antisense oligonucleotide treatments
David Cheerie, Margaret M. Meserve, Danique Beijer, Charu Kaiwar, ... Jack T. Morgan, ... Rosan Lechner, ... Bianca Zardetto, ... Annemieke Aartsma-Rus, ... Marlen C. Lauffer, the N=1 Collaborative
The American Journal of Human Genetics (2025), https://doi.org/10.1016/j.ajhg.2025.02.017
Preclinical assessment of splicing modulation therapy for ABCA4 variant c.768G>T in Stargardt disease
Dyah W. Karjosukarso, Femke Bukkems, Lonneke Duijkers, Tomasz Z. Tomkiewicz, Julia Kiefmann, Andrei Sarlea, Sander Bervoets, Irene Vázquez-Domínguez, Laurie L. Molday, Robert S. Molday, Mihai G. Netea, Carel B. Hoyng, Alejandro Garanto & Rob W. J. Collin
Communications Medicine volume 5, Article number: 25 (2025)
ExonViz: A website and Python package to visualize transcripts and genetic variants
Redmar R. van den Berg, Marlen C. Lauffer, Jeroen F.J. Laros
medRxiv 2024.09.18.24313945;
https://doi.org/10.1101/2024.09.18.24313945
Annemieke Aartsma-Rus, Rob W.J. Collin, Ype Elgersma, Marlen C. Lauffer, Willeke van Roon-Mom
Practical Recommendations for the Selection of Patients for Individualized Splice-Switching ASO-Based Treatments
Bianca Zardetto, Marlen C. Lauffer, Willeke van Roon-Mom and Annemieke Aartsma-Rus
Possibilities and limitations of antisense oligonucleotide therapeutics for the treatment of monogenic disorders
Marlen C. Lauffer, Willeke van Roon-Mom and Annemieke Aartsma-Rus
Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1 Exon Skipping Antisense Oligonucleotides
Annemieke Aartsma-Rus, Alejandro Garanto, Willeke van Roon-Mom, Erin M. McConnell, Victoria Suslovitch, Winston X. Yan, Jonathan K. Watts, and Timothy W. Yu, on behalf of the N=1 Collaborative
Development of tailored splice switching oligonucleotides for progressive brain disorders in Europe: development, regulation and implementation considerations
Annemieke Aartsma-Rus, Willeke van Roon-Mom, Marlen Lauffer, Christine Siezen, Britt Duijndam, Tineke Coenen-de Roo, Rebecca Schüle, Matthis Synofzik, and Holm Graessner
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
Willeke van Roon-Mom, Chantal Ferguson and Annemieke Aartsma-Rus
Therapeutic Strategies for Spinocerebellar Ataxia Type 1
Laurie M. C. Kerkhof, Bart P. C. van de Warrenburg, Willeke M.C. van Roon-Mom and Ronald A. M. Buijsen