Zobrazeno 1 - 10
of 22
pro vyhledávání: '"Susannah Patarroyo-White"'
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
Autor:
Arjan van der Flier, Zhan Liu, Siyuan Tan, Kai Chen, Douglas Drager, Tongyao Liu, Susannah Patarroyo-White, Haiyan Jiang, David R Light
Publikováno v:
PLoS ONE, Vol 10, Iss 4, p e0124930 (2015)
We recently developed a longer lasting recombinant factor VIII-Fc fusion protein, rFVIIIFc, to extend the half-life of replacement FVIII for the treatment of people with hemophilia A. In order to elucidate the biological mechanism for the elongated h
Externí odkaz:
https://doaj.org/article/851241490ca94f369db6a73e66bcee0e
Autor:
Yang Buyue, Tongyao Liu, John D Kulman, Garabet G Toby, George D Kamphaus, Susannah Patarroyo-White, Qi Lu, Thomas J Reidy, Baisong Mei, Haiyan Jiang, Glenn F Pierce, Jurg M Sommer, Robert T Peters
Publikováno v:
PLoS ONE, Vol 9, Iss 11, p e113600 (2014)
Recombinant factor VIII Fc (rFVIIIFc) is a fusion protein consisting of a single B-domain-deleted (BDD) FVIII linked recombinantly to the Fc domain of human IgG1 to extend half-life. To determine if rFVIIIFc could be further improved by maintaining t
Externí odkaz:
https://doaj.org/article/9f7063df33694e378018959bd4c66d30
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
Autor:
Michela Milani, Tongyao Liu, Andrea Annoni, Luigi Naldini, Cesare Canepari, Rosalia Curto, Fabio Russo, Susannah Patarroyo-White, Chiara Brombin, Paola Albertini, Christian Mueller, Tiziana Plati, Eduard Ayuso, Alessio Cantore, Ilaria Visigalli, Mauro Biffi, Douglas Drager
Liver gene therapy with adeno-associated viral (AAV) vectors delivering a clotting factor transgene into hepatocytes has shown multi-year therapeutic benefit in adults with hemophilia. However, anti-AAV pre-existing immunity and the mostly episomal n
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::842361699de168ed14f6b3f49cd7fd94
https://doi.org/10.21203/rs.3.rs-858195/v1
https://doi.org/10.21203/rs.3.rs-858195/v1
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
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
Autor:
Haiyan Jiang, Arjan van der Flier, David R. Light, Kai Chen, Susannah Patarroyo-White, Zhan Liu, Siyuan Tan, Tongyao Liu, Douglas Drager
Publikováno v:
PLoS ONE
PLoS ONE, Vol 10, Iss 4, p e0124930 (2015)
PLoS ONE, Vol 10, Iss 4, p e0124930 (2015)
We recently developed a longer lasting recombinant factor VIII-Fc fusion protein, rFVIIIFc, to extend the half-life of replacement FVIII for the treatment of people with hemophilia A. In order to elucidate the biological mechanism for the elongated h
Autor:
Dea Nagy, Glenn Pierce, Joseph A. Vargas, Tongyao Liu, Ciaran Scallan, Jurg M. Sommer, Haiyan Jiang, Sharmila Vijay, Federico Mingozzi, Linda B. Couto, Shangzhen Zhou, Katherine A. High, Susannah Patarroyo-White
Publikováno v:
Blood. 108:3321-3328
In a clinical study of recombinant adeno-associated virus-2 expressing human factor IX (AAV2-FIX), we detected 2 impediments to long-term gene transfer. First, preexisting anti-AAV neutralizing antibodies (NABs) prevent vector from reaching the targe
Autor:
Kay S. Arnold, Susannah Patarroyo-White, Lucia Rohrer, Thomas L. Innerarity, Maureen E. Balestra, Martin Hersberger, Xiaobing Qian
Publikováno v:
Biochemical Journal. 369:255-262
Apolipoprotein (apo) B mRNA editing is the deamination of C6666 to uridine, which results in translation of the apoB-48 protein instead of the genomically encoded apoB-100. ApoB-48-containing lipoproteins are cleared more rapidly from plasma and are
Publikováno v:
Journal of Neurochemistry. 76:1308-1314
Recently, two dinucleotide deletions were detected in the mRNA of the amyloid precursor protein (APP) from cerebral cortex neurons of patients with sporadic Alzheimer's disease (AD) or Down's syndrome. These deletions resulted in truncation of APP, p