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.
Dissemination
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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 and Publications
Posters 2023
Posters 2022
PUBLICATIONS
Possibilities and limitations of antisense oligonucleotide therapeutics for the treatment of monogenic disorders
Marlen C. Lauffer, Willeke van Roon-Mom and Annemieke Aartsma-Rus
Communications Medicine, 2024, 4:6.
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
Nucleic Acid Therapeutics, 2023, 33(1), 17-25.
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
RNA, 2023 Jan 20;rna.079540.122. doi: 10.1261/rna.079540.122.
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
Nucleic Acid Ther, 2023, 33(4), 234-237.
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
Biomolecules, 2023, 13(5), 788.
‘N of 1’ therapies need a better model
Annemieke Aartsma-Rus
Nature Medicine, June 2021, VOL 27 ,939.