The MID1 Protein: A Promising Therapeutic Target in Huntington’s Disease

por Heinz, Annika, Schilling, Judith, van Roon-Mom, Willeke, Krauß, Sybille
Publicado 2021

Ataxin-3 Protein and RNA Toxicity in Spinocerebellar Ataxia Type 3: Current Insights and Emerging Therapeutic Strategies

por Evers, Melvin M., Toonen, Lodewijk J. A., van Roon-Mom, Willeke M. C.
Publicado 2013

Antisense Oligonucleotide-Mediated Removal of the Polyglutamine Repeat in Spinocerebellar Ataxia Type 3 Mice

por Toonen, Lodewijk J.A., Rigo, Frank, van Attikum, Haico, van Roon-Mom, Willeke M.C.
Publicado 2017

Juvenile‐Onset Huntington Disease Pathophysiology and Neurodevelopment: A Review

por Bakels, Hannah S., Roos, Raymund A.C., van Roon‐Mom, Willeke M.C., de Bot, Susanne T.
Publicado 2021

Antisense oligonucleotide-mediated exon skipping as a strategy to reduce proteolytic cleavage of ataxin-3

por Toonen, Lodewijk J. A., Schmidt, Iris, Luijsterburg, Martijn S., van Attikum, Haico, van Roon-Mom, Willeke M. C.
Publicado 2016

Genetics, Mechanisms, and Therapeutic Progress in Polyglutamine Spinocerebellar Ataxias

por Buijsen, Ronald A.M., Toonen, Lodewijk J.A., Gardiner, Sarah L., van Roon-Mom, Willeke M.C.
Publicado 2019

Antisense-Mediated RNA Targeting: Versatile and Expedient Genetic Manipulation in the Brain

por Zalachoras, Ioannis, Evers, Melvin M., van Roon-Mom, Willeke M. C., Aartsma-Rus, Annemieke M., Meijer, Onno C.
Publicado 2011

Increased sensitivity of next generation sequencing-based expression profiling after globin reduction in human blood RNA

por Mastrokolias, Anastasios, den Dunnen, Johan T, van Ommen, GertJan B, 't Hoen, Peter AC, van Roon-Mom, Willeke MC
Publicado 2012

Statistical method for modeling sequencing data from different technologies in longitudinal studies with application to Huntington disease

por Fuady, Angga M., van Roon‐Mom, Willeke M. C., Kiełbasa, Szymon M., Uh, Hae‐Won, Houwing‐Duistermaat, Jeanine J.
Publicado 2020

Therapeutic Strategies for Spinocerebellar Ataxia Type 1

por Kerkhof, Laurie M. C., van de Warrenburg, Bart P. C., van Roon-Mom, Willeke M. C., Buijsen, Ronald A. M.
Publicado 2023

Exploring the Transcriptome of Ciliated Cells Using In Silico Dissection of Human Tissues

por Ivliev, Alexander E., 't Hoen, Peter A. C., van Roon-Mom, Willeke M. C., Peters, Dorien J. M., Sergeeva, Marina G.
Publicado 2012

Osteopontin and phospho‐SMAD2/3 are associated with calcification of vessels in D‐CAA, an hereditary cerebral amyloid angiopathy

por Grand Moursel, Laure, van der Graaf, Linda M., Bulk, Marjolein, van Roon‐Mom, Willeke M.C., van der Weerd, Louise
Publicado 2019

Preparing n-of-1 Antisense Oligonucleotide Treatments for Rare Neurological Diseases in Europe: Genetic, Regulatory, and Ethical Perspectives

por Synofzik, Matthis, van Roon-Mom, Willeke M.C, Marckmann, Georg, van Duyvenvoorde, Hermine A., Graessner, Holm, Schüle, Rebecca, Aartsma-Rus, Annemieke
Publicado 2022

Post‐mortem 7T MR imaging and neuropathology in middle stage juvenile‐onset Huntington disease: A case report

por Bakels, Hannah S., van Duinen, Sjoerd G., de Bresser, Jeroen, van Roon‐Mom, Willeke M. C., van der Weerd, Louise, de Bot, Susanne T.
Publicado 2022

Cost-effective HRMA pre-sequence typing of clone libraries; application to phage display selection

por Pepers, Barry A, Schut, Menno H, Vossen, Rolf HAM, van Ommen, Gert-Jan B, den Dunnen, Johan T, van Roon-Mom, Willeke MC
Publicado 2009

Intracerebroventricular Administration of a 2′-O-Methyl Phosphorothioate Antisense Oligonucleotide Results in Activation of the Innate Immune System in Mouse Brain

por Toonen, Lodewijk J.A., Casaca-Carreira, João, Pellisé-Tintoré, Maria, Mei, Hailiang, Temel, Yasin, Jahanshahi, Ali, van Roon-Mom, Willeke M.C.
Publicado 2018

Human Induced Pluripotent Stem Cell-Based Modelling of Spinocerebellar Ataxias

por Hommersom, Marina P., Buijsen, Ronald A. M., van Roon-Mom, Willeke M. C., van de Warrenburg, Bart P. C., van Bokhoven, Hans
Publicado 2021

Development of tailored splice-switching oligonucleotides for progressive brain disorders in Europe: development, regulation, and implementation considerations

por Aartsma-Rus, Annemieke, van Roon-Mom, Willeke, Lauffer, Marlen, Siezen, Christine, Duijndam, Britt, Coenen-de Roo, Tineke, Schüle, Rebecca, Synofzik, Matthis, Graessner, Holm
Publicado 2023

Common disease signatures from gene expression analysis in Huntington’s disease human blood and brain

por Mina, Eleni, van Roon-Mom, Willeke, Hettne, Kristina, van Zwet, Erik, Goeman, Jelle, Neri, Christian, A.C. ’t Hoen, Peter, Mons, Barend, Roos, Marco
Publicado 2016

Pathological characterization of T2*-weighted MRI contrast in the striatum of Huntington’s disease patients

por Bulk, Marjolein, Hegeman-Kleinn, Ingrid, Kenkhuis, Boyd, Suidgeest, Ernst, van Roon-Mom, Willeke, Lewerenz, Jan, van Duinen, Sjoerd, Ronen, Itamar, van der Weerd, Louise
Publicado 2020