Preclinical assessment of an optimized AAV-FVIII vector in mice and non-human primates for the treatment of hemophilia A

Autor: Liron Elkouby, Sean M. Armour, Raffaella Toso, Marti DiPietro, Robert J. Davidson, Giang N. Nguyen, Mallory Willet, Stephanie Kutza, Joseph Silverberg, Jennifer Frick, Marco Crosariol, Yuhuan Wang, Chuansong Wang, Katherine A. High, Denise E. Sabatino, Xavier M. Anguela

Publikováno v: Molecular Therapy: Methods & Clinical Development, Vol 24, Iss , Pp 20-29 (2022)

Extensive clinical data from liver-mediated gene therapy trials have shown that dose-dependent immune responses against the vector capsid may impair or even preclude transgene expression if not managed successfully with prompt immune suppression. The

Externí odkaz: https://doaj.org/article/8d15f9b5838e44d49c75621a886fa6f5

IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies

Autor: Adeline Miranda, Dan Lupo, Federico Mingozzi, Xavier M. Anguela, Joseph Silverberg, Karen Huang, Heena Beck, Saghana Muraleetharan, Béatrice Marolleau, Fanny Collaud, Laetitia van Wittengerghe, Sean M. Armour, Christian Leborgne, Elena Barbon, Sandrine Delignat, Jeffrey M. Alexander, Hayley Hanby, Victoria Daventure, Giuseppe Ronzitti, Sébastien Lacroix-Desmazes, Anna Fabiano, Daniel M. Cohen

Publikováno v: Nature Medicine
Nature Medicine, Nature Publishing Group, 2020, 26 (7), pp.1096-1101. ⟨10.1038/s41591-020-0911-7⟩
Nature Medicine, 2020, 26 (7), pp.1096-1101. ⟨10.1038/s41591-020-0911-7⟩

Neutralizing antibodies to adeno-associated virus (AAV) vectors are highly prevalent in humans1,2, and block liver transduction3–5 and vector readministration6; thus, they represent a major limitation to in vivo gene therapy. Strategies aimed at ov

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ff04724b6af379a7e9370adc25a9aa43
https://hal.archives-ouvertes.fr/hal-03031762