Zobrazeno 1 - 5
of 5
pro vyhledávání: '"Judith C T van Deutekom"'
Autor:
Nicole A Datson, Anchel González-Barriga, Eleni Kourkouta, Rudie Weij, Jeroen van de Giessen, Susan Mulders, Outi Kontkanen, Taneli Heikkinen, Kimmo Lehtimäki, Judith C T van Deutekom
Publikováno v:
PLoS ONE, Vol 12, Iss 2, p e0171127 (2017)
The aim of these studies was to demonstrate the therapeutic capacity of an antisense oligonucleotide with the sequence (CUG)7 targeting the expanded CAG repeat in huntingtin (HTT) mRNA in vivo in the R6/2 N-terminal fragment and Q175 knock-in Hunting
Externí odkaz:
https://doaj.org/article/bca1853fe95b4f42b79763185181a38e
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:
Anchel González-Barriga, Julia Kranzen, Huib J E Croes, Suzanne Bijl, Walther J A A van den Broek, Ingeborg D G van Kessel, Baziel G M van Engelen, Judith C T van Deutekom, Bé Wieringa, Susan A M Mulders, Derick G Wansink
Publikováno v:
PLoS ONE, Vol 10, Iss 3, p e0121556 (2015)
Myotonic Dystrophy type 1 (DM1) is a multisystemic disease caused by toxic RNA from a DMPK gene carrying an expanded (CTG•CAG)n repeat. Promising strategies for treatment of DM1 patients are currently being tested. These include antisense oligonucl
Externí odkaz:
https://doaj.org/article/e315422256e242feba27e3e2c97a4a55
Autor:
Ingrid E C Verhaart, Laura van Vliet-van den Dool, Jessica A Sipkens, Sjef J de Kimpe, Ingrid G M Kolfschoten, Judith C T van Deutekom, Lia Liefaard, Jim E Ridings, Steve R Hood, Annemieke Aartsma-Rus
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 3, Iss C (2014)
Antisense-mediated exon skipping is currently in clinical development for Duchenne muscular dystrophy (DMD) to amend the consequences of the underlying genetic defect and restore dystrophin expression. Due to turnover of compound, transcript, and pro
Externí odkaz:
https://doaj.org/article/381c115381eb4ba695493ef95aa99b17
Autor:
Melvin M Evers, Barry A Pepers, Judith C T van Deutekom, Susan A M Mulders, Johan T den Dunnen, Annemieke Aartsma-Rus, Gert-Jan B van Ommen, Willeke M C van Roon-Mom
Publikováno v:
PLoS ONE, Vol 6, Iss 9, p e24308 (2011)
To date there are 9 known diseases caused by an expanded polyglutamine repeat, with the most prevalent being Huntington's disease. Huntington's disease is a progressive autosomal dominant neurodegenerative disorder for which currently no therapy is a
Externí odkaz:
https://doaj.org/article/8fcae1147e504440b8318728c2011151