Zobrazeno 1 - 6
of 6
pro vyhledávání: '"Elisabeth A. Spronck"'
Autor:
Elisabeth A. Spronck, Ying Poi Liu, Jacek Lubelski, Erich Ehlert, Sander Gielen, Paula Montenegro-Miranda, Martin de Haan, Bart Nijmeijer, Valerie Ferreira, Harald Petry, Sander J. van Deventer
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 15, Iss , Pp 221-231 (2019)
Gene therapy for severe hemophilia B is advancing and offers sustained disease amelioration with a single treatment. We have reported the efficacy and safety of AMT-060, an investigational gene therapy comprising an adeno-associated virus serotype 5
Externí odkaz:
https://doaj.org/article/f68597eceef64b579989937b741d7460
Autor:
Elisabeth A. Spronck, Cynthia C. Brouwers, Astrid Vallès, Martin de Haan, Harald Petry, Sander J. van Deventer, Pavlina Konstantinova, Melvin M. Evers
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 13, Iss , Pp 334-343 (2019)
Huntington disease (HD) is a fatal neurodegenerative disorder caused by an autosomal dominant CAG repeat expansion in the huntingtin (HTT) gene. The translated expanded polyglutamine repeat in the HTT protein is known to cause toxic gain of function.
Externí odkaz:
https://doaj.org/article/80df46c476b84f378c89927542f2b137
Autor:
Elisabeth A. Spronck, Astrid Vallès, Margit H. Lampen, Paula S. Montenegro-Miranda, Sonay Keskin, Liesbeth Heijink, Melvin M. Evers, Harald Petry, Sander J. van Deventer, Pavlina Konstantinova, Martin de Haan
Publikováno v:
Brain Sciences, Vol 11, Iss 2, p 129 (2021)
Huntington disease (HD) is a fatal, neurodegenerative genetic disorder with aggregation of mutant Huntingtin protein (mutHTT) in the brain as a key pathological mechanism. There are currently no disease modifying therapies for HD; however, HTT-loweri
Externí odkaz:
https://doaj.org/article/003bb3f8cb854143be21f0cb86c10923
Autor:
Pavlina Konstantinova, Martin de Haan, Anouk Stam, Astrid Vallès, Carlos Vendrell-Tornero, Rienk Nieuwland, Sander J. H. van Deventer, Elisabeth A. Spronck, Sonay Keskin, Melvin M. Evers, Jolanda Snapper, Jana Miniarikova, Marina Sogorb-Gonzalez
Publikováno v:
Brain Communications
Brain Communications, 3(2). Oxford University Press
Brain Communications, 3(2). Oxford University Press
The preclinical development of microRNA-based gene therapies for inherited neurodegenerative diseases is accompanied by translational challenges. Due to the inaccessibility of the brain to periodically evaluate therapy effects, accessible and reliabl
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::e820d92c6a1850456c1b027565f9b149
https://doi.org/10.1093/braincomms/fcab054
https://doi.org/10.1093/braincomms/fcab054
Autor:
Paula S Montenegro-Miranda, Valerie Ferreira, Harald Petry, Elisabeth A. Spronck, Sander J. H. van Deventer, Bart A. Nijmeijer, Erich Ehlert, Sander Gielen, Jacek Lubelski, Ying Poi Liu, Martin de Haan
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 15, Iss, Pp 221-231 (2019)
Molecular Therapy. Methods & Clinical Development
Molecular Therapy-Methods and Clinical Development, 15, 221-231. CELL PRESS
Molecular Therapy. Methods & Clinical Development
Molecular Therapy-Methods and Clinical Development, 15, 221-231. CELL PRESS
Gene therapy for severe hemophilia B is advancing and offers sustained disease amelioration with a single treatment. We have reported the efficacy and safety of AMT-060, an investigational gene therapy comprising an adeno-associated virus serotype 5
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::e4b4294973adf70abce05445843ac809
https://doi.org/10.1016/j.omtm.2019.09.005
https://doi.org/10.1016/j.omtm.2019.09.005
Autor:
Pavlina Konstantinova, Elisabeth A. Spronck, Cynthia Brouwers, Melvin M. Evers, Martin de Haan, Astrid Vallès, Sander J. H. van Deventer, Harald Petry
Publikováno v:
Molecular Therapy-Methods and Clinical Development, 13, 334-343. CELL PRESS
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 13, Iss, Pp 334-343 (2019)
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 13, Iss, Pp 334-343 (2019)
Huntington disease (HD) is a fatal neurodegenerative disorder caused by an autosomal dominant CAG repeat expansion in the huntingtin (HTT) gene. The translated expanded polyglutamine repeat in the HTT protein is known to cause toxic gain of function.
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::aa3b564939eca57a4b425ae5bb8c85ab
http://hdl.handle.net/1887/3196166
http://hdl.handle.net/1887/3196166