Zobrazeno 1 - 10
of 34
pro vyhledávání: '"Gwladys Gernoux"'
Autor:
Marina Zieger, Florie Borel, Cynthia Greer, Gwladys Gernoux, Meghan Blackwood, Terence R. Flotte, Christian Mueller
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 25, Iss , Pp 425-438 (2022)
α1-antitrypsin deficiency is a rare genetic condition that can cause liver and/or lung disease. There is currently no cure for this disorder, although repeated infusions of plasma-purified protein may slow down emphysema progression. Gene therapy in
Externí odkaz:
https://doaj.org/article/dd33fcbe12cd471e89896bea164e7f7b
Autor:
Samer Masri, Laure Carré, Nicolas Jaulin, Céline Vandamme, Célia Couzinié, Aurélien Guy-Duché, Jean-Baptiste Dupont, Allwyn Pereira, Eric Charpentier, Laurent David, Gwladys Gernoux, Mickaël Guilbaud, Oumeya Adjali
Publikováno v:
International Journal of Molecular Sciences, Vol 24, Iss 13, p 10447 (2023)
Recombinant Adeno-Associated Virus (rAAV) is considered as one of the most successful and widely used viral vectors for in vivo gene therapy. However, host immune responses to the vector and/or the transgene product remain a major hurdle to successfu
Externí odkaz:
https://doaj.org/article/fe3eaffab875485da755793c0229386f
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:
Alisha M. Gruntman, Gwladys Gernoux, Qiushi Tang, Guo-Jie Ye, Dave R. Knop, Gensheng Wang, Janet Benson, Kristen E. Coleman, Allison M. Keeler, Christian Mueller, Louis G. Chicoine, Jeffrey D. Chulay, Terence R. Flotte
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 13, Iss , Pp 233-242 (2019)
Phase 1 and phase 2 gene therapy trials using intramuscular (IM) administration of a recombinant adeno-associated virus serotype 1 (rAAV1) for replacement of serum alpha-1 antitrypsin (AAT) deficiency have shown long-term (5-year) stable transgene ex
Externí odkaz:
https://doaj.org/article/ece0ba0412444a99888e9c8c1783ad84
Autor:
Terence R. Flotte, Oguz Cataltepe, Ajit Puri, Ana Rita Batista, Richard Moser, Diane McKenna-Yasek, Catherine Douthwright, Gwladys Gernoux, Meghan Blackwood, Christian Mueller, Phillip W. L. Tai, Xuntian Jiang, Scot Bateman, Spiro G. Spanakis, Julia Parzych, Allison M. Keeler, Aly Abayazeed, Saurabh Rohatgi, Laura Gibson, Robert Finberg, Bruce A. Barton, Zeynep Vardar, Mohammed Salman Shazeeb, Matthew Gounis, Cynthia J. Tifft, Florian S. Eichler, Robert H. Brown, Douglas R. Martin, Heather L. Gray-Edwards, Miguel Sena-Esteves
Publikováno v:
Nature Medicine. 28:251-259
Tay-Sachs disease (TSD) is an inherited neurological disorder caused by deficiency of hexosaminidase A (HexA). Here, we describe an adeno-associated virus (AAV) gene therapy expanded-access trial in two patients with infantile TSD (IND 18225) with sa
Autor:
Virginie Pichard, Oumeya Adjali, Nicolas Jaulin, Malo Journou, Johanne Le Duff, Gwladys Gernoux, Adrien Leger, Caroline Le Guiner, Yan Cherel, Marie Devaux, Mickaël Guilbaud, Jack-Yves Deschamps, Philippe Moullier
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 20, Iss, Pp 660-674 (2021)
Molecular Therapy-Methods and Clinical Development
Molecular Therapy-Methods and Clinical Development, Nature Publishing Group, 2021, 20, pp.660-674. ⟨10.1016/j.omtm.2021.02.003⟩
Molecular Therapy. Methods & Clinical Development
Molecular Therapy-Methods and Clinical Development
Molecular Therapy-Methods and Clinical Development, Nature Publishing Group, 2021, 20, pp.660-674. ⟨10.1016/j.omtm.2021.02.003⟩
Molecular Therapy. Methods & Clinical Development
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
Autor:
Christian Mueller, Gwladys Gernoux, Jeffrey Teckman, Annamaria Carissimo, Edoardo Nusco, Nicola Brunetti-Pierri, Pasquale Piccolo, Sergio Attanasio, Rosa Ferriero, Rossella De Cegli, Severo Campione
Publikováno v:
Journal of Biological Chemistry. 295:13213-13223
α1-Antitrypsin (AAT) encoded by the SERPINA1 gene is an acute-phase protein synthesized in the liver and secreted into the circulation. Its primary role is to protect lung tissue by inhibiting neutrophil elastase. The Z allele of SERPINA1 encodes a
Autor:
Nicholas Wightman, Merit Cudkowicz, Margaret A. Owegi, Dario Gelevski, Gwladys Gernoux, Meghan Blackwood, Christian Mueller, James D. Berry, Sarah Luppino, Catherine Douthwright, Robert H. Brown, Terrence R. Flotte, Derek H. Oakley, Diane McKenna-Yasek, Lindsay Pothier, Matthew P. Frosch
Publikováno v:
New England Journal of Medicine. 383:151-158
Two patients with familial amyotrophic lateral sclerosis (ALS) and mutations in the gene encoding superoxide dismutase 1 (SOD1) were treated with a single intrathecal infusion of adeno-associated virus encoding a microRNA targeting SOD1. In Patient 1
Autor:
Gwladys Gernoux, Marina Zieger, Terence R. Flotte, Christian Mueller, Alisha M. Gruntman, Meghan Blackwood
Publikováno v:
Molecular Therapy
With the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) approvals for Zolgensma, Luxturna, and Glybera, recombinant adeno-associated viruses (rAAVs) are considered efficient tools for gene transfer. However, studies in anim
Autor:
Marina, Zieger, Florie, Borel, Cynthia, Greer, Gwladys, Gernoux, Meghan, Blackwood, Terence R, Flotte, Christian, Mueller
Publikováno v:
Molecular therapy. Methodsclinical development. 25
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