Zobrazeno 1 - 7
of 7
pro vyhledávání: '"Guillaume Precigout"'
Autor:
Christina Gedicke‐Hornung, Verena Behrens‐Gawlik, Silke Reischmann, Birgit Geertz, Doreen Stimpel, Florian Weinberger, Saskia Schlossarek, Guillaume Précigout, Ingke Braren, Thomas Eschenhagen, Giulia Mearini, Stéphanie Lorain, Thomas Voit, Patrick A. Dreyfus, Luis Garcia, Lucie Carrier
Publikováno v:
EMBO Molecular Medicine, Vol 5, Iss 7, Pp 1128-1145 (2013)
Abstract Exon skipping mediated by antisense oligoribonucleotides (AON) is a promising therapeutic approach for genetic disorders, but has not yet been evaluated for cardiac diseases. We investigated the feasibility and efficacy of viral‐mediated A
Externí odkaz:
https://doaj.org/article/2151f46ca23e4c23b5b19581f81c7297
Autor:
Giulia Mearini, Doreen Stimpel, Elisabeth Krämer, Birgit Geertz, Ingke Braren, Christina Gedicke-Hornung, Guillaume Précigout, Oliver J Müller, Hugo A Katus, Thomas Eschenhagen, Thomas Voit, Luis Garcia, Stéphanie Lorain, Lucie Carrier
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 2, Iss C (2013)
RNA trans-splicing has been explored as a therapeutic option for a variety of genetic diseases, but not for cardiac genetic disease. Hypertrophic cardiomyopathy (HCM) is an autosomal-dominant disease, characterized by left ventricular hypertrophy (LV
Externí odkaz:
https://doaj.org/article/71f14212fc7c4a9d880522573ae986ba
Autor:
Gabriella Dias Florencio, Guillaume Precigout, Cyriaque Beley, Pierre-Olivier Buclez, Luis Garcia, Rachid Benchaouir
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 2, Iss , Pp - (2015)
Recombinant adeno-associated viruses (rAAV) are promising candidates for gene therapy approaches. The last two decades were particularly fruitful in terms of processes applied in the production and purification of this type of gene transfer vectors.
Externí odkaz:
https://doaj.org/article/4b292448e03f4d25996b5f5abceaeb4b
Autor:
Thomas Voit, Stéphanie Lorain, Aurélie Goyenvalle, Guillaume Precigout, Luis Garcia, Maëva Le Hir, Kay E. Davies, Cécile Peccate
Publikováno v:
Molecular Therapy. 21(8):1551-1558
In the context of future adeno-associated viral (AAV)-based clinical trials for Duchenne myopathy, AAV genome fate in dystrophic muscles is of importance considering the viral capsid immunogenicity that prohibits recurring treatments. We showed that
Autor:
Graziella Griffith, Guillaume Precigout, Stéphanie Lorain, Luis Garcia, Thomas Voit, Maëva Le Hir, Susanne Philippi, Arnaud Jollet, Cécile Peccate, Kamel Mamchaoui
Publikováno v:
Nucleic Acids Research
RNA-based therapeutic approaches using splice-switching oligonucleotides have been successfully applied to rescue dystrophin in Duchenne muscular dystrophy (DMD) preclinical models and are currently being evaluated in DMD patients. Although the modul
Autor:
Luis Garcia, Guillaume Precigout, Cyriaque Beley, Pierre-Olivier Buclez, Gabriella Dias Florencio, Rachid Benchaouir
Publikováno v:
Molecular Therapy. Methods & Clinical Development
Molecular Therapy-Methods and Clinical Development
Molecular Therapy-Methods and Clinical Development, Nature Publishing Group, 2015, 2, pp.15024. ⟨10.1038/mtm.2015.24⟩
Molecular Therapy: Methods & Clinical Development, Vol 2, Iss, Pp-(2015)
Molecular Therapy-Methods and Clinical Development, 2015, 2, pp.15024. ⟨10.1038/mtm.2015.24⟩
Molecular Therapy-Methods and Clinical Development
Molecular Therapy-Methods and Clinical Development, Nature Publishing Group, 2015, 2, pp.15024. ⟨10.1038/mtm.2015.24⟩
Molecular Therapy: Methods & Clinical Development, Vol 2, Iss, Pp-(2015)
Molecular Therapy-Methods and Clinical Development, 2015, 2, pp.15024. ⟨10.1038/mtm.2015.24⟩
Recombinant adeno-associated viruses (rAAV) are promising candidates for gene therapy approaches. The last two decades were particularly fruitful in terms of processes applied in the production and purification of this type of gene transfer vectors.
Autor:
Cécile Peccate, Luis Garcia, Kay E. Davies, M. Le Hir, Thomas Voit, Stéphanie Lorain, Aurélie Goyenvalle, Guillaume Precigout
Publikováno v:
Neuromuscular Disorders. 23:841
In the context of future AAV-based clinical trials for Duchenne myopathy, AAV genome fate in dystrophic muscles is of importance considering the viral capsid immunogenicity that prohibits recurring treatments. We followed AAV genome copy numbers afte