Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor VIII activity in non-human primates

Autor: Michela Milani, Cesare Canepari, Tongyao Liu, Mauro Biffi, Fabio Russo, Tiziana Plati, Rosalia Curto, Susannah Patarroyo-White, Douglas Drager, Ilaria Visigalli, Chiara Brombin, Paola Albertini, Antonia Follenzi, Eduard Ayuso, Christian Mueller, Andrea Annoni, Luigi Naldini, Alessio Cantore

Publikováno v: Nature Communications, Vol 13, Iss 1, Pp 1-14 (2022)

“Lentiviral gene therapy to the liver establishes stable long-term normal to supra-normal coagulation factor VIII activity in mouse models of hemophilia A and in non-human primates, representing a potential new treatment option for people with hemo

Externí odkaz: https://doaj.org/article/c9b97a0d651b468fbb352c8d225f07b9

BIVV001, a new class of factor VIII replacement for hemophilia A that is independent of von Willebrand factor in primates and mice

Autor: Joe Salas, Tongyao Liu, Randy Mauldin, Jiayun Liu, Terrence M. Dobrowsky, Jurg M. Sommer, Volker Schellenberger, Susannah Patarroyo-White, Mark Tie, Ayman Ismail, Buyue Yang, Haiyan Jiang, Arjan van der Flier, Qi Lu, Chris Furcht, Nancy Moore, Tyler Carlage, John Kulman, Baisong Mei, Amy M Holthaus, Allison Goodman, Zhan Liu, Lily Zhu, Robert T. Peters, Deana Rabinovich, Glenn F. Pierce, Ekta Seth Chhabra, Douglas Drager, Oblaise Mercury, Zhiqian Liu

Publikováno v: Blood

Factor VIII (FVIII) replacement products enable comprehensive care in hemophilia A. Treatment goals in severe hemophilia A are expanding beyond low annualized bleed rates to include long-term outcomes associated with high sustained FVIII levels. Endo

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::e5b05ed2ddd184e92d73acf24aec1f10
https://doi.org/10.1182/blood.2019001292

Phagocytosis-shielded lentiviral vectors improve liver gene therapy in nonhuman primates

Autor: Mauro Biffi, Daniela Cesana, Federica Moalli, Tongyao Liu, Matteo Iannacone, Douglas Drager, Patrizia Cristofori, Sara Bartolaccini, Fabio Russo, Andrea Raimondi, Alessio Cantore, Ilaria Visigalli, Robert T. Peters, Eugenio Montini, Andrea Calabria, Michela Milani, Susannah Patarroyo-White, Andrea Annoni, Eduard Ayuso, Luigi Naldini

Publikováno v: Science Translational Medicine
Science Translational Medicine, American Association for the Advancement of Science, 2019, 11 (493), pp.eaav7325. ⟨10.1126/scitranslmed.aav7325⟩
Science Translational Medicine, American Association for the Advancement of Science (AAAS), 2019, 11 (493), pp.eaav7325. ⟨10.1126/scitranslmed.aav7325⟩
Sci Transl Med

International audience; Liver-directed gene therapy for the coagulation disorder hemophilia showed safe and effective results in clinical trials using adeno-associated viral vectors to replace a functional coagulation factor, although some unmet need

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::6649d598dd8d5d1cee1da1118caa1bc3
https://doi.org/10.1126/scitranslmed.aav7325

Prolonged activity of a recombinant factor VIII-Fc fusion protein in hemophilia A mice and dogs

Autor: Cara Fraley, Justin McCue, Xin Zhang, Timothy C. Nichols, Elizabeth P. Merricks, Helen W. G. Franck, Thomas Reidy, George D. Kamphaus, Glenn F. Pierce, Susan C. Low, Jennifer A. Dumont, Alan J. Bitonti, Tongyao Liu, Haiyan Jiang, Paul Sakorafas, Douglas Drager

Publikováno v: Blood. 119(13)

Despite proven benefits, prophylactic treatment for hemophilia A is hampered by the short half-life of factor VIII. A recombinant factor VIII-Fc fusion protein (rFVIIIFc) was constructed to determine the potential for reduced frequency of dosing. rFV

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::1c5750ffec372f36445fb77ccef78014
https://pubmed.ncbi.nlm.nih.gov/22246033

Different Contribution of Hematopoietic and Somatic Cell Types Expressing FcRn to the Prolonged Half-Life of Recombinant Factor VIII Fc and Recombinant FIX Fc

Autor: David R. Light, Kai Chen, Haiyan Jiang, Douglas Drager, Arjan van der Flier, Siyuan Tan, Zhan Liu, Keith Wharton, Sebastien Vallee

Publikováno v: Blood. 120:1103-1103

Abstract 1103 Introduction: Recombinant fusion of a single molecule of either FVIII or FIX to the Fc domain of IgG1 (rFVIIIFc or rFIXFc, respectively) has been shown to decrease clearance compared to rFIX or rFVIII in an FcRn-dependent manner (Blood

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::865085aedf6de40dc0bcd1e5fb37b056
https://doi.org/10.1182/blood.v120.21.1103.1103