Block or degrade? Balancing on- and off-target effects of antisense strategies against transcripts with expanded triplet repeats in DM1

Autor: Najoua El Boujnouni, M. Leontien van der Bent, Marieke Willemse, Peter A.C. ’t Hoen, Roland Brock, Derick G. Wansink

Publikováno v: Molecular Therapy: Nucleic Acids, Vol 32, Iss , Pp 622-636 (2023)

Antisense oligonucleotide (ASO) therapies for myotonic dystrophy type 1 (DM1) are based on elimination of transcripts containing an expanded repeat or inhibition of sequestration of RNA-binding proteins. This activity is achievable by both degradatio

Externí odkaz: https://doaj.org/article/97e890b2b5784324b32954702b47d821

Inhibition of microRNA-494-3p activates Wntsignaling and reduces proinflammatorymacrophage polarization in atherosclerosis

Autor: Eva van Ingen, Amanda C. Foks, Tamar Woudenberg, M. Leontien van der Bent, Alwin de Jong, Philipp J. Hohensinner, Johann Wojta, Ilze Bot, Paul.H.A. Quax, Anne Yaël Nossent

Publikováno v: Molecular Therapy: Nucleic Acids, Vol 26, Iss , Pp 1228-1239 (2021)

We have previously shown that treatment with third-generation antisense oligonucleotides against miR-494-3p (3GA-494) reduces atherosclerotic plaque progression and stabilizes lesions, both in early and established plaques, with reduced macrophage co

Externí odkaz: https://doaj.org/article/3c732ba897314891bf7651163691b08a

Functional Intercellular Transmission of miHTT via Extracellular Vesicles: An In Vitro Proof-of-Mechanism Study

Autor: Roberto D. V. S. Morais, Marina Sogorb-González, Citlali Bar, Nikki C. Timmer, M. Leontien Van der Bent, Morgane Wartel, Astrid Vallès

Publikováno v: Cells, Vol 11, Iss 17, p 2748 (2022)

Huntington’s disease (HD) is a fatal neurodegenerative disorder caused by GAG expansion in exon 1 of the huntingtin (HTT) gene. AAV5-miHTT is an adeno-associated virus serotype 5-based vector expressing an engineered HTT-targeting microRNA (miHTT).

Externí odkaz: https://doaj.org/article/34d98b50c74a4ed08dd0502971363f9c

C/D box snoRNA SNORD113-6/AF357425 plays a dual role in integrin signalling and arterial fibroblast function via pre-mRNA processing and 2'O-ribose methylation

Autor: M. Leontien van der Bent, Hailiang Mei, Reinier A. Boon, Daphne A L van den Homberg, Paul H.A. Quax, Johann Wojta, Veerle Kremer, Nikolina Papac-Milicevic, A. Yaël Nossent, Eva van Ingen

Publikováno v: van Ingen, E, van den Homberg, D A L, van der Bent, M L, Mei, H, Papac-Milicevic, N, Kremer, V, Boon, R A, Quax, P H A, Wojta, J & Nossent, A Y 2022, ' C/D box snoRNA SNORD113-6/AF357425 plays a dual role in integrin signalling and arterial fibroblast function via pre-mRNA processing and 2'O-ribose methylation ', Human Molecular Genetics, vol. 31, no. 7, pp. 1051-1066 . https://doi.org/10.1093/hmg/ddab304
Human Molecular Genetics, 31(7), 1051-1066. OXFORD UNIV PRESS
Human Molecular Genetics, 31(7), 1051-1066. Oxford University Press

We have previously shown that C/D box small nucleolar RNAs (snoRNAs) transcribed from the DLK1-DIO3 locus on human chromosome 14 (14q32) are associated with cardiovascular disease. DLK1-DIO3 snoRNAs are ‘orphan snoRNAs’ that have no known targets

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::e2b58e08eb44713352dff1899c53be85
https://research.vumc.nl/en/publications/6e40b26c-732c-409a-ae7e-ad9a7fce5582

C/D box snoRNA SNORD113-6 guides 2'-O-methylation and protects against site-specific fragmentation of tRNA

Autor: Eva, van Ingen, Pleun A M, Engbers, Tamar, Woudenberg, M Leontien, van der Bent, Hailiang, Mei, Johann, Wojta, Paul H A, Quax, A Yaël, Nossent

Publikováno v: Molecular therapy. Nucleic acids. 30

C/D box small nucleolar RNAs (snoRNAs) of the DLK1-DIO3 locus are associated with vascular remodeling and cardiovascular disease. None of these snoRNAs has any known targets yet except for one, AF357425/SNORD113-6. We previously showed that this snoR

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid________::8db9a1c40b9173139f9028af9fe707b8
https://pubmed.ncbi.nlm.nih.gov/36250206