Repeated dosing of AAV-mediated liver gene therapy in juvenile rat and mouse models of Crigler-Najjar syndrome type I

Autor: Shi, Xiaoxia, Bortolussi, Giulia, Collaud, Fanny, Lebrun, Pierre-Romain, Bloemendaal, Lysbeth ten, Guerchet, Nicolas, Rudi de Waart, Dirk, Sellier, Pauline, Duijst, Suzanne, Veron, Philippe, Mingozzi, Federico, Kishimoto, Takashi Kei, Ronzitti, Giuseppe, Bosma, Piter, Muro, Andrés F.

Publikováno v: In Molecular Therapy - Methods & Clinical Development 12 December 2024 32(4)

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Development of a dual hybrid AAV vector for endothelial-targeted expression of von Willebrand factor

Autor: Barbon, Elena, Kawecki, Charlotte, Marmier, Solenne, Sakkal, Aboud, Collaud, Fanny, Charles, Severine, Ronzitti, Giuseppe, Casari, Caterina, Christophe, Olivier D., Denis, Cécile V., Lenting, Peter J., Mingozzi, Federico

Publikováno v: Gene Therapy; April 2023, Vol. 30 Issue: 3-4 p245-254, 10p

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

Autor: Collaud, Fanny, Bortolussi, Giulia, Guianvarc'h, Laurence, Aronson, Sem, Bordet, Thierry, Veron, Philippe, Charles, Severine, Vidal, Patrice, Sola, Marcelo Simon, Rundwasser, Stephanie, Dufour, Delphine, Lacoste, Florence, Luc, Cyril, Wittenberghe, Laetitia, Martin, Samia, Le Bec, Christine, Bosma, Piter, Muro, Andres, Ronzitti, Giuseppe, Hebben, Matthias, Mingozzi, Federico, Guianvarc’h, Laurence

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

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