Marlen Lauffer
@lauffermarlen
Lover of rare diseases, especially neurological disorders. Working on developing individualized ASO treatments at #DCRT, together with #1M1M, and #N1C.
ID: 1169332058927304709
http://rnatherapy.nl 04-09-2019 19:31:20
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Had the pleasure to join the #GERS2024 today and talk about our work at the #DCRT and N1 Collaborative on how we can identify eligible candidates for N=1 therapies. Great to hear and see what Genomics England is working on, and am especially excited about their Generation Study.
Our paper on the DCRT is out now in Therapeutic Advances in Rare Disease Joining forces to develop individualized antisense oligonucleotides for patients with brain or eye diseases: the example of the Dutch Center for RNA Therapeutics - (sagepub.com)
Thank you to our colleagues from UK Platform for Nucleic Acid Therapies- UPNAT for your participation!
Thanks to AnnemiekeAartsma-Rus for having our ExonViz tool featured in #apaperaday! I regularly use ExonViz to explain exon skipping to patients and families and for all my presentations.
At the #OTS2024 this week and we have two of our early career reaserchers from the DCRT Leiden presenting their work. Jack will talk about exon skipping for protein tandem repeat domains and Raúl will present on his ASO tox studies in mice and rats! AnnemiekeAartsma-Rus @roon_mom
