Výsledky vyhledávání - "Charles, Severine"

Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome

Publikováno v: Molecular Therapy-Methods and Clinical Development
Molecular Therapy-Methods and Clinical Development, Nature Publishing Group, 2019, 12, pp.157-174. ⟨10.1016/j.omtm.2018.12.011⟩
Molecular Therapy-Methods & Clinical Development
Molecular Therapy-Methods and Clinical Development, 2019, 12, pp.157-174. ⟨10.1016/j.omtm.2018.12.011⟩
Molecular Therapy. Methods & Clinical Development
Molecular therapy. Methods & clinical development, 12, 157-174. Nature Publishing Group
Molecular Therapy-Methods & Clinical Development, 2019, 12, pp.157-174. ⟨10.1016/j.omtm.2018.12.011⟩
Molecular Therapy: Methods & Clinical Development, Vol 12, Iss, Pp 157-174 (2019)

Adeno-associated viruses (AAVs) are among the most efficient vectors for liver gene therapy. Results obtained in the first hemophilia clinical trials demonstrated the long-term efficacy of this approach in humans, showing efficient targeting of hepat

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::15c64845ecf5191c292eb40b9a420e80
https://www.scopus.com/inward/record.uri?partnerID=HzOxMe3b&scp=85059833634&origin=inward

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Long-term correction of ornithine transcarbamylase deficiency in Spf-Ash mice with a translationally optimized AAV vector

Autor: Sola, Marcelo Simon, Kishimoto, Takashi Kei, De Sabbata, Giulia, Boisgérault, Florence, Guarnaccia, Corrado, Iaconcig, Alessandra, Bortolussi, Giulia, Collaud, Fanny, Ronzitti, Giuseppe, Sola, Marcelo, Vidal, Patrice, Rouillon, Jeremy, Charles, Severine, Nicastro, Emanuele, D’Antiga, Lorenzo, Ilyinskii, Petr, Mingozzi, Federico, Muro, Andrés

Publikováno v: Molecular Therapy-Methods and Clinical Development
Molecular Therapy-Methods and Clinical Development, Nature Publishing Group, 2021, 20, pp.169-180. ⟨10.1016/j.omtm.2020.11.005⟩
Molecular Therapy-Methods and Clinical Development, 2021, 20, pp.169-180. ⟨10.1016/j.omtm.2020.11.005⟩
Molecular Therapy: Methods & Clinical Development, Vol 20, Iss, Pp 169-180 (2021)
Molecular Therapy. Methods & Clinical Development

Ornithine transcarbamylase deficiency (OTCD) is an X-linked liver disorder caused by partial or total loss of OTC enzyme activity. It is characterized by elevated plasma ammonia, leading to neurological impairments, coma, and death in the most severe

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a696612410ed50b494a5c222c5224075
https://hal.sorbonne-universite.fr/hal-03193757/document

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Single‐domain antibodies targeting antithrombin reduce bleeding in hemophilic mice with or without inhibitors

Autor: Barbon, Elena, Ayme, Gabriel, Mohamadi, Amel, Ottavi, Jean-François, Kawecki, Charlotte, Casari, Caterina, Verhenne, Sebastien, Marmier, Solenne, Van Wittenberghe, Laetitia, Charles, Severine, Collaud, Fanny, Denis, Cecile, Christophe, Olivier, Mingozzi, Federico, Lenting, Peter

Publikováno v: EMBO Molecular Medicine
EMBO Molecular Medicine, Wiley Open Access, 2020, 12 (4), pp.e11298. ⟨10.15252/emmm.201911298⟩
EMBO Molecular Medicine, 2020, 12 (4), pp.e11298. ⟨10.15252/emmm.201911298⟩
EMBO Molecular Medicine, Vol 12, Iss 4, Pp n/a-n/a (2020)

Novel therapies for hemophilia, including non‐factor replacement and in vivo gene therapy, are showing promising results in the clinic, including for patients having a history of inhibitor development. Here, we propose a novel therapeutic approach

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid_dedup__::f2124e0bff36528e9ada978918252631
https://www.hal.inserm.fr/inserm-02551254

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