Zobrazeno 1 - 10
of 173
pro vyhledávání: '"Philippe, Moullier"'
Autor:
Mathieu Mével, Virginie Pichard, Mohammed Bouzelha, Dimitri Alvarez-Dorta, Pierre-Alban Lalys, Nathalie Provost, Marine Allais, Alexandra Mendes, Elodie Landagaray, Jean-Baptiste Ducloyer, Estelle Toublanc, Anne Galy, Nicole Brument, Gaëlle M. Lefevre, Sébastien G. Gouin, Carolina Isiegas, Guylène Le Meur, Thérèse Cronin, Caroline Le Guiner, Michel Weber, Philippe Moullier, Eduard Ayuso, David Deniaud, Oumeya Adjali
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 32, Iss 1, Pp 101187- (2024)
Inherited retinal diseases are a leading and untreatable cause of blindness and are therefore candidate diseases for gene therapy. Recombinant vectors derived from adeno-associated virus (rAAV) are currently the most promising vehicles for in vivo th
Externí odkaz:
https://doaj.org/article/1edecadd12924319b147b4cab1a426f8
Autor:
Caroline Le Guiner, Xiao Xiao, Thibaut Larcher, Aude Lafoux, Corinne Huchet, Gilles Toumaniantz, Oumeya Adjali, Ignacio Anegon, Séverine Remy, Josh Grieger, Juan Li, Vahid Farrokhi, Hendrik Neubert, Jane Owens, Maritza McIntyre, Philippe Moullier, R. Jude Samulski
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 30, Iss , Pp 30-47 (2023)
Duchenne muscular dystrophy (DMD) is an X-linked disease caused by loss-of-function mutations in the dystrophin gene and is characterized by muscle wasting and early mortality. Adeno-associated virus-mediated gene therapy is being investigated as a t
Externí odkaz:
https://doaj.org/article/7a89e1515ecf4452944c4318d3851f25
Autor:
Gwladys Gernoux, Mickaël Guilbaud, Marie Devaux, Malo Journou, Virginie Pichard, Nicolas Jaulin, Adrien Léger, Johanne Le Duff, Jack-Yves Deschamps, Caroline Le Guiner, Philippe Moullier, Yan Cherel, Oumeya Adjali
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 20, Iss , Pp 660-674 (2021)
Adeno-associated virus (AAV) vectors are considered efficient vectors for gene transfer, as illustrated by recent successful clinical trials targeting retinal or neurodegenerative disorders. However, limitations as host immune responses to AAV capsid
Externí odkaz:
https://doaj.org/article/38d74ee6093e4b7fa86f324ccc982a8e
Autor:
Karim Bey, Johan Deniaud, Laurence Dubreil, Béatrice Joussemet, Joseph Cristini, Carine Ciron, Juliette Hordeaux, Morwenn Le Boulc’h, Kevin Marche, Maud Maquigneau, Michaël Guilbaud, Rosalie Moreau, Thibaut Larcher, Jack-Yves Deschamps, Marion Fusellier, Véronique Blouin, Caroline Sevin, Nathalie Cartier, Oumeya Adjali, Patrick Aubourg, Philippe Moullier, Marie-Anne Colle
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 17, Iss , Pp 771-784 (2020)
The identification of the most efficient method for whole central nervous system targeting that is translatable to humans and the safest route of adeno-associated virus (AAV) administration is a major concern for future applications in clinics. Addit
Externí odkaz:
https://doaj.org/article/a0aedb1aca064422b991e72f09f1cc8d
Autor:
Céline Vandamme, Rebecca Xicluna, Leslie Hesnard, Marie Devaux, Nicolas Jaulin, Mickaël Guilbaud, Johanne Le Duff, Célia Couzinié, Philippe Moullier, Xavier Saulquin, Oumeya Adjali
Publikováno v:
Frontiers in Immunology, Vol 10 (2020)
Pre-existing immunity to AAV capsid may compromise the safety and efficiency of rAAV-mediated gene transfer in patients. Anti-capsid cytotoxic immune responses have proven to be a challenge to characterize because of the scarcity of circulating AAV-s
Externí odkaz:
https://doaj.org/article/21c2793766ba4036b7fafebbf31d8574
Autor:
Achille François, Mohammed Bouzelha, Emilie Lecomte, Frédéric Broucque, Magalie Penaud-Budloo, Oumeya Adjali, Philippe Moullier, Véronique Blouin, Eduard Ayuso
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 10, Iss , Pp 223-236 (2018)
Although the clinical use of recombinant adeno-associated virus (rAAV) vectors is constantly increasing, the development of suitable quality control methods is still needed for accurate vector characterization. Among the quality criteria, the titrati
Externí odkaz:
https://doaj.org/article/e86bc233d9c34383bc042649c09abbea
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
Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy
Autor:
Caroline Le Guiner, Laurent Servais, Marie Montus, Thibaut Larcher, Bodvaël Fraysse, Sophie Moullec, Marine Allais, Virginie François, Maeva Dutilleul, Alberto Malerba, Taeyoung Koo, Jean-Laurent Thibaut, Béatrice Matot, Marie Devaux, Johanne Le Duff, Jack-Yves Deschamps, Inès Barthelemy, Stéphane Blot, Isabelle Testault, Karim Wahbi, Stéphane Ederhy, Samia Martin, Philippe Veron, Christophe Georger, Takis Athanasopoulos, Carole Masurier, Federico Mingozzi, Pierre Carlier, Bernard Gjata, Jean-Yves Hogrel, Oumeya Adjali, Fulvio Mavilio, Thomas Voit, Philippe Moullier, George Dickson
Publikováno v:
Nature Communications, Vol 8, Iss 1, Pp 1-15 (2017)
Duchenne muscular dystrophy is a progressive degenerative disease of muscles caused by mutations in the dystrophin gene. Here the authors use AAV vectors to deliver microdystrophin to dogs with muscular dystrophy, and show restoration of dystrophin e
Externí odkaz:
https://doaj.org/article/d425f7f6bf364aa99c6850ae61ecdd6b
Autor:
Audrey Bourdon, Virginie François, Liwen Zhang, Aude Lafoux, Bodvael Fraysse, Gilles Toumaniantz, Thibaut Larcher, Tiphaine Girard, Mireille Ledevin, Cyrielle Lebreton, Agnès Hivonnait, Anna Creismeas, Marine Allais, Basile Marie, Justine Guguin, Véronique Blouin, Séverine Remy, Ignacio Anegon, Corinne Huchet, Alberto Malerba, Betty Kao, Anita Le Heron, Philippe Moullier, George Dickson, Linda Popplewell, Oumeya Adjali, Federica Montanaro, Caroline Le Guiner
Publikováno v:
Gene Therapy. 29:520-535
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::3d9c1614fc49440d97af2761d82421d2
https://doi.org/10.1038/s41434-022-00317-6
https://doi.org/10.1038/s41434-022-00317-6
Autor:
Mathieu Mével, Virginie Pichard, Mohammed Bouzelha, Dimitri Alvarez-Dorta, Pierre-Alban Lalys, Nathalie Provost, Marine Allais, Alexandra Mendes, Elodie Landagaray, Jean-Baptiste Ducloyer, Anne Galy, Nicole Brument, Gaëlle M. Lefevre, Sébastien G. Gouin, Carolina Isiegas, Guylène Le Meur, Thérèse Cronin, Caroline Le Guiner, Michel Weber, Philippe Moullier, Eduard Ayuso, David Deniaud, Oumeya Adjali
Inherited retinal diseases are a leading and untreatable cause of blindness and are therefore candidate diseases for gene therapy. Recombinant vectors derived from adeno-associated virus (rAAV) are currently the most promising vehicles forin vivother
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::754177853388fd915b92c4ee4b3e16c7
https://doi.org/10.1101/2022.12.01.518481
https://doi.org/10.1101/2022.12.01.518481
