Zobrazeno 1 - 4
of 4
pro vyhledávání: '"Marina Sogorb-González"'
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
Autor:
Josse A. Depla, Marina Sogorb-Gonzalez, Lance A. Mulder, Vivi M. Heine, Pavlina Konstantinova, Sander J. van Deventer, Katja C. Wolthers, Dasja Pajkrt, Adithya Sridhar, Melvin M. Evers
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 18, Iss , Pp 167-175 (2020)
The development of gene therapies for central nervous system disorders is challenging because it is difficult to translate preclinical data from current in vitro and in vivo models to the clinic. Therefore, we developed induced pluripotent stem cell
Externí odkaz:
https://doaj.org/article/6801bff942ae41f78c9545db8a90bbd9
Autor:
Sonay Keskin, Cynthia C. Brouwers, Marina Sogorb-Gonzalez, Raygene Martier, Josse A. Depla, Astrid Vallès, Sander J. van Deventer, Pavlina Konstantinova, Melvin M. Evers
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 15, Iss , Pp 275-284 (2019)
Huntington disease (HD) is a fatal neurodegenerative genetic disorder, thought to reflect a toxic gain of function in huntingtin (Htt) protein. Adeno-associated viral vector serotype 5 (AAV5)- microRNA targeting huntingtin (miHTT) is a HD gene-therap
Externí odkaz:
https://doaj.org/article/ea22fa55f85944d198f8e622dca8256f
Autor:
Raygene Martier, Marina Sogorb-Gonzalez, Janice Stricker-Shaver, Jeannette Hübener-Schmid, Sonay Keskin, Jiri Klima, Lodewijk J. Toonen, Stefan Juhas, Jana Juhasova, Zdenka Ellederova, Jan Motlik, Eva Haas, Sander van Deventer, Pavlina Konstantinova, Huu Phuc Nguyen, Melvin M. Evers
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 15, Iss , Pp 343-358 (2019)
Spinocerebellar ataxia type 3 (SCA3), or Machado-Joseph disease (MJD), is a progressive neurodegenerative disorder caused by a CAG expansion in the ATXN3 gene. The expanded CAG repeat is translated into a prolonged polyglutamine repeat in the ataxin-
Externí odkaz:
https://doaj.org/article/9b77ea2f73b84aa0bf0e991ed509f3df