Zobrazeno 1 - 10
of 1 802
pro vyhledávání: '"Aartsma-Rus A"'
Publikováno v:
The Application of Clinical Genetics, Vol 2014, Iss default, Pp 245-252 (2014)
Virginia Arechavala-Gomeza,1 Bernard Khoo,2 Annemieke Aartsma-Rus3 1Neuromuscular Disorders Group, BioCruces Health Research Institute, Barakaldo, Bizkaia, Spain; 2Endocrinology, Division of Medicine, University College London, London, UK; 3Departmen
Externí odkaz:
https://doaj.org/article/1cfe420a25504a6b9feb705ade14dfa4
Publikováno v:
Communications Medicine, Vol 4, Iss 1, Pp 1-11 (2024)
Abstract Antisense oligonucleotides (ASOs) are incredibly versatile molecules that can be designed to specifically target and modify RNA transcripts to slow down or halt rare genetic disease progression. They offer the potential to target groups of p
Externí odkaz:
https://doaj.org/article/5921451646084845bc63da2e688a2b76
Publikováno v:
RNA Biology, Vol 20, Iss 1, Pp 693-702 (2023)
Mutations in the DMD gene are causative for Duchenne muscular dystrophy (DMD). Antisense oligonucleotide (AON) mediated exon skipping to restore disrupted dystrophin reading frame is a therapeutic approach that allows production of a shorter but func
Externí odkaz:
https://doaj.org/article/24bc75579f454439b33a70bce5092238
Autor:
David Cheerie, Marlen Lauffer, Danique Beijer, Matthis Synofzik, Annemieke Aartsma-Rus, Michael Szego, Kimberly Amburgey, Brian Kalish, James Dowling, Gregory Costain
Publikováno v:
Genetics in Medicine Open, Vol 2, Iss , Pp 101068- (2024)
Externí odkaz:
https://doaj.org/article/da13f9ba7b74444b8558dcca41060f10
Autor:
Zalachoras Ioannis, Grootaers Gwendolynn, van Weert Lisa TCM, Aubert Yves, de Kreij Suzanne R, Datson Nicole A, van Roon-Mom Willeke MC, Aartsma-Rus Annemieke, Meijer Onno C
Publikováno v:
BMC Neuroscience, Vol 14, Iss 1, p 5 (2013)
Abstract Background Antisense oligonucleotide (AON)-mediated exon skipping is a powerful tool to manipulate gene expression. In the present study we investigated the potential of exon skipping by local injection in the central nucleus of the amygdala
Externí odkaz:
https://doaj.org/article/b15f33767e234ce599b06ed3d6884d44
Akademický článek
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Autor:
van Ommen Gert Jan B, den Dunnen Johan T, de Gorter David JJ, Terlouw Samuel, van Heiningen Sandra H, Hoogaars Willem MH, Kemaladewi Dwi U, Aartsma-Rus Annemieke, ten Dijke Peter, 't Hoen Peter AC
Publikováno v:
BMC Medical Genomics, Vol 4, Iss 1, p 36 (2011)
Abstract Background Myostatin is a potent muscle growth inhibitor that belongs to the Transforming Growth Factor-β (TGF-β) family. Mutations leading to non functional myostatin have been associated with hypermuscularity in several organisms. By con
Externí odkaz:
https://doaj.org/article/07a8f75a00944b1382c2e811e51b786e
Autor:
van Ommen Gert-Jan B, van Deutekom Judith CT, de Winter Christa L, van Vliet Laura, Aartsma-Rus Annemieke
Publikováno v:
BMC Medical Genetics, Vol 9, Iss 1, p 105 (2008)
Abstract Background The specific skipping of an exon, induced by antisense oligonucleotides (AON) during splicing, has shown to be a promising therapeutic approach for Duchenne muscular dystrophy (DMD) patients. As different mutations require skippin
Externí odkaz:
https://doaj.org/article/44560fc3edca4f8286524152a4724532
Publikováno v:
BMC Medical Genetics, Vol 8, Iss 1, p 43 (2007)
Abstract Background Antisense-mediated exon skipping is currently one of the most promising therapeutic approaches for Duchenne muscular dystrophy (DMD). Using antisense oligonucleotides (AONs) targeting specific exons the DMD reading frame is restor
Externí odkaz:
https://doaj.org/article/0b9d8287a04a4b24b13ac23e081d3dc1
Akademický článek
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K zobrazení výsledku je třeba se přihlásit.
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