Zobrazeno 1 - 9
of 9
pro vyhledávání: '"Ruurd C. Verheul"'
Autor:
Monika Hiller, Maria Sofia Falzarano, Iker Garcia-Jimenez, Valentina Sardone, Ruurd C Verheul, Linda Popplewell, Karen Anthony, Estibaliz Ruiz-Del-Yerro, Hana Osman, Jelle J Goeman, Kamel Mamchaoui, George Dickson, Alessandra Ferlini, Francesco Muntoni, Annemieke Aartsma-Rus, Virginia Arechavala-Gomeza, Nicole A Datson, Pietro Spitali
Publikováno v:
PLoS ONE, Vol 13, Iss 10, p e0204485 (2018)
BACKGROUND:Duchenne muscular dystrophy is a lethal disease caused by lack of dystrophin. Skipping of exons adjacent to out-of-frame deletions has proven to restore dystrophin expression in Duchenne patients. Exon 51 has been the most studied target i
Externí odkaz:
https://doaj.org/article/bfcda443e99b4e5388c172844550fbbe
Publikováno v:
PLoS ONE, Vol 11, Iss 9, p e0162467 (2016)
Antisense oligonucleotides (AONs) in clinical development for Duchenne muscular dystrophy (DMD) aim to induce skipping of a specific exon of the dystrophin transcript during pre-mRNA splicing. This results in restoration of the open reading frame and
Externí odkaz:
https://doaj.org/article/4ef94e21a9cf40918ef1c1568d290745
Autor:
Melissa Mulder, Ruurd C. Verheul, Judith C.T. van Deutekom, Bas Groenendaal, Nicole A. Datson, Eleni Kourkouta, Anchel González-Barriga, Sieto Bosgra, Jukka Puoliväli, Jussi Toivanen, Rudie Weij
Publikováno v:
Molecular Therapy. Nucleic Acids
Molecular Therapy: Nucleic Acids, Vol 17, Iss, Pp 601-614 (2019)
Molecular Therapy: Nucleic Acids, Vol 17, Iss, Pp 601-614 (2019)
Spinocerebellar ataxia type 3 (SCA3) and type 1 (SCA1) are dominantly inherited neurodegenerative disorders that are currently incurable. Both diseases are caused by a CAG-repeat expansion in exon 10 of the Ataxin-3 and exon 8 of the Ataxin-1 gene, r
Autor:
Annemieke Aartsma-Rus, Begoña Aguilera, Ruurd C. Verheul, Christa L Tanganyika-de Winter, Valeriano Diaz Parillas, Silvana M.G. Jirka, Peter C de Visser, Peter A C 't Hoen
Publikováno v:
Molecular Therapy
Molecular Therapy, 26, 1, pp. 132-147
Molecular Therapy, 26(1), 132-147
Molecular Therapy, 26, 132-147
Molecular Therapy, 26, 1, pp. 132-147
Molecular Therapy, 26(1), 132-147
Molecular Therapy, 26, 132-147
Duchenne muscular dystrophy (DMD) is a severe, progressive muscle wasting disorder caused by reading frame disrupting mutations in the DMD gene. Exon skipping is a therapeutic approach for DMD. It employs antisense oligonucleotides (AONs) to restore
Autor:
Nicole A. Datson, Pietro Spitali, Kamel Mamchaoui, Karen Anthony, Monika Hiller, Alessandra Ferlini, E. Ruiz-Del-Yerro, H. Osman, Linda Popplewell, George Dickson, Maria Sofia Falzarano, Ruurd C. Verheul, Valentina Sardone, Jelle J. Goeman, I. Garcia-Jimenez, Francesco Muntoni, Annemieke Aartsma-Rus, Virginia Arechavala-Gomeza
Publikováno v:
PLoS ONE
PLoS ONE, Public Library of Science, 2018, 13 (10), pp.e0204485. ⟨10.1371/journal.pone.0204485⟩
PLoS ONE, Vol 13, Iss 10, p e0204485 (2018)
PLoS ONE, 13(10)
PLoS ONE, Public Library of Science, 2018, 13 (10), pp.e0204485. ⟨10.1371/journal.pone.0204485⟩
PLoS ONE, Vol 13, Iss 10, p e0204485 (2018)
PLoS ONE, 13(10)
Background: Duchenne muscular dystrophy is a lethal disease caused by lack of dystrophin. Skipping of exons adjacent to out-of-frame deletions has proven to restore dystrophin expression in Duchenne patients. Exon 51 has been the most studied target
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c02b36021a375ce634dd2ecbce9e33d2
https://hal.sorbonne-universite.fr/hal-03285360
https://hal.sorbonne-universite.fr/hal-03285360
Publikováno v:
PLoS ONE, Vol 11, Iss 9, p e0162467 (2016)
PLoS ONE
PLoS ONE
Antisense oligonucleotides (AONs) in clinical development for Duchenne muscular dystrophy (DMD) aim to induce skipping of a specific exon of the dystrophin transcript during pre-mRNA splicing. This results in restoration of the open reading frame and
Autor:
Ingrid G.M. Kolfschoten, Ruurd C. Verheul, J.C.T. van Deutekom, P.C. de Visser, Rudie Weij, A. Baghat, M.M. Plug, J.A.M. Janson, R.E.Y. van den Eijnde
Publikováno v:
Neuromuscular Disorders. 24:820-821
Antisense oligonucleotides (AONs) for Duchenne muscular dystrophy (DMD) are designed to induce skipping of a single exon during pre-mRNA splicing, thus restoring the transcript’s open reading frame and consequently synthesis of the deficient dystro
Publikováno v:
Neuromuscular Disorders. 24:820
Antisense oligonucleotide (AON) therapies for Duchenne muscular dystrophy (DMD) are aimed at inducing skipping of specific exons during pre-mRNA splicing. This results in restoration of the reading frame and consequently synthesis of a shorter yet fu
Autor:
Masja de Haas, Yixian Zhao, Nigel M. Stapleton, Stefania P. Bjarnarson, Ruurd C. Verheul, Inger Sandlie, Ingileif Jonsdottir, Annette M. Stemerding, Jan Terje Andersen, Jorrit Gerritsen, Gestur Vidarsson, C. Ellen van der Schoot, Marion Kleijer
Publikováno v:
Nature Communications
Nature communications, 2. Nature Publishing Group
Nature communications, 2. Nature Publishing Group
Human IgG3 displays the strongest effector functions of all IgG subclasses but has a short half-life for unresolved reasons. Here we show that IgG3 binds to IgG-salvage receptor (FcRn), but that FcRn-mediated transport and rescue of IgG3 is inhibited