Zobrazeno 1 - 7
of 7
pro vyhledávání: '"Claire Domenger"'
Autor:
Claire Domenger, Marine Allais, Virginie François, Adrien Léger, Emilie Lecomte, Marie Montus, Laurent Servais, Thomas Voit, Philippe Moullier, Yann Audic, Caroline Le Guiner
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 10, Iss , Pp 277-291 (2018)
Non-coding uridine-rich small nuclear RNAs (UsnRNAs) have emerged in recent years as effective tools for exon skipping for the treatment of Duchenne muscular dystrophy (DMD), a degenerative muscular genetic disorder. We recently showed the high capac
Externí odkaz:
https://doaj.org/article/b56772ff2df9496ca4c2cf486504309a
Autor:
Claire Domenger, Dirk Grimm
Publikováno v:
Human Molecular Genetics. 28:R3-R14
Recombinant adeno-associated viruses (AAV) are under intensive investigation in numerous clinical trials after they have emerged as a highly promising vector for human gene therapy. Best exemplifying their power and potential is the authorization of
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::98cdd948fe487c6593273b35ef971da4
https://doi.org/10.1093/hmg/ddz148
https://doi.org/10.1093/hmg/ddz148
Autor:
Claire Domenger, Manuel Mastel, Kathleen Börner, Roland Eils, Mareike D. Hoffmann, Stefanie Grosse, Dirk Grimm, Sabine Aschenbrenner, Dominik Niopek, Julia Fakhiri, Kleopatra Rapti
Publikováno v:
Nucleic Acids Research
The rapid development of CRISPR–Cas technologies brought a personalized and targeted treatment of genetic disorders into closer reach. To render CRISPR-based therapies precise and safe, strategies to confine the activity of Cas(9) to selected cells
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::6d39d3dfe399f9b663df0a4ff74a7e03
https://pubmed.ncbi.nlm.nih.gov/30982889
https://pubmed.ncbi.nlm.nih.gov/30982889
Autor:
Dominik Niopek, Julia Fakhiri, Kleopatra Rapti, Roland Eils, Mareike D. Hoffmann, Stefanie Grosse, Dirk Grimm, Manuel Mastel, Sabine Aschenbrenner, Claire Domenger
The rapid development of CRISPR/Cas technologies brought a personalized and targeted treatment of genetic disorders into closer reach. To render CRISPR-based therapies precise and safe, strategies to confine the activity of Cas(9) to selected cells a
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a1ddf2aff217adaaa9a51612f6cf3e6e
Autor:
Thomas Voit, M. Allais, Marie Montus, Caroline Le Guiner, Yann Audic, Adrien Leger, Emilie Lecomte, Laurent Servais, Philippe Moullier, Claire Domenger, Virginie François
Publikováno v:
Molecular Therapy-Nucleic Acids
Molecular Therapy-Nucleic Acids, Elsevier, 2018, 10, pp.277-291. ⟨10.1016/j.omtn.2017.12.008⟩
Molecular Therapy-Nucleic Acids, 2018, 10, pp.277-291. ⟨10.1016/j.omtn.2017.12.008⟩
Molecular Therapy: Nucleic Acids, Vol 10, Iss, Pp 277-291 (2018)
Molecular Therapy. Nucleic Acids
Molecular Therapy-Nucleic Acids, Elsevier, 2018, 10, pp.277-291. ⟨10.1016/j.omtn.2017.12.008⟩
Molecular Therapy-Nucleic Acids, 2018, 10, pp.277-291. ⟨10.1016/j.omtn.2017.12.008⟩
Molecular Therapy: Nucleic Acids, Vol 10, Iss, Pp 277-291 (2018)
Molecular Therapy. Nucleic Acids
International audience; Non-coding uridine-rich small nuclear RNAs (UsnRNAs) have emerged in recent years as effective tools for exon skipping for the treatment of Duchenne muscular dystrophy (DMD), a degenerative muscular genetic disorder. We recent
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::972ce6eff761ffa91ac2666b5bab1696
https://www.hal.inserm.fr/inserm-01734756/document
https://www.hal.inserm.fr/inserm-01734756/document
Autor:
Philippe Moullier, M. Allais, Claire Domenger, L. Servais, Marie Montus, Caroline Le Guiner, Virginie François, Thomas Voit, Aurélie Lardenois
Publikováno v:
Molecular Therapy. 23
Duchenne muscular dystrophy (DMD) is a severe muscle wasting disorder affecting 1 male in 5000 and caused by mutations in the gene encoding for the Dystrophin protein. So far, there are no strongly effective treatments but gene-based therapies are cu
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::499bb67c77aa51d57c53dd387511d441
Autor:
Yan Cherel, Philippe Veron, F. Barnay-Toutain, Virginie Latournerie, Christian Leborgne, Jean-Laurent Thibaud, Marie Montus, Claire Wary, Nicolas Jaulin, C. Le Guiner, Claire Domenger, Christel Rivière, Oumeya Adjali, H. Goubin, T. Voit, Carole Masurier, Lydie Guigand, N. Delaunay, Philippe Moullier, Sylvie Boutin, Diana Desgue, B. Matot, Gisèle Bonne, Virginie François, J.Y. Hogrel, Maeva Dutilleul, L. Servais, Pierre G. Carlier, Marie Devaux, M. Allais, Sophie Moullec, J. Le Duff, Mickaël Guilbaud, Jack-Yves Deschamps, T. Larche
Publikováno v:
Neuromuscular Disorders. 24:822
We have previously demonstrated that a recombinant adeno-associated virus vector, serotype 8 (rAAV8) carrying a modified U7snRNA sequence promoting exon skipping injected in GRMD by locoregional transvenous perfusion of the forelimb, restores up to 8
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::0892a6fbcc4080858cffd4ff57690e21
https://doi.org/10.1016/j.nmd.2014.06.106
https://doi.org/10.1016/j.nmd.2014.06.106