Article
Immunology
Dorien De Pooter, Ellen Van Gulck, Antony Chen, Claire F. Evans, Jean-Marc Neefs, Helen Horton, Daniel Boden
Summary: Despite the availability of a prophylactic vaccine for HBV for over 30 years, nearly 300 million people are still chronically infected worldwide. Chronic HBV patients exhibit weak CD8(+) T-cell responses, but a therapeutic DNA vaccine has shown to induce robust immune responses in both healthy and infected animal models.
Article
Medicine, Research & Experimental
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
Summary: This article introduces the gene therapy for hemophilia A and validates the feasibility of reducing the viral vector dose to minimize the risk of immune response through preclinical studies.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT
(2022)
Article
Medicine, General & Internal
Haneesha Mohan, Monica Guzman Lenis, Evelyn Y. Laurette, Oscar Tejada, Tanvi Sanghvi, Kit-Yi Leung, Lindsay S. Cahill, John G. Sled, Paul Delgado-Olguin, Nicholas D. E. Greene, Andrew J. Copp, Lena Serghides
Summary: DTG at therapeutic doses may increase the risk of fetal defects, especially NTDs, while the relationship between DTG at subtherapeutic doses and fetal anomalies is unclear.
Article
Immunology
A. Nguyen, Y. Repesse, M. Ebbo, Y. Allenbach, O. Benveniste, J. M. Vallat, L. Magy, S. Deshayes, G. Maigne, H. de Boysson, A. Karnam, S. Delignat, S. Lacroix-Desmazes, J. Bayry, A. Aouba
Summary: The mechanisms of action of IVIg in autoimmune diseases are not fully understood, but may be related to the over-production of IL-11, which increases platelets, von Willebrand factor, and FVIII levels.
CLINICAL AND EXPERIMENTAL IMMUNOLOGY
(2021)
Article
Hematology
Melissa Bou-Jaoudeh, Sandrine Delignat, Victoria Daventure, Jan Astermark, Herve Levesque, Jordan Dimitrov, Claire Deligne, Valerie Proulle, Sebastien Lacroix-Desmazes
Summary: Neutralizing anti-factor VIII (FVIII) antibodies, known as FVIII inhibitors, pose a major hindrance to replacement therapy for hemophilia A patients. Imlifidase (IdeS), an endopeptidase, has shown potential in eliminating FVIII inhibitors in both vitro and mouse models. By cleaving anti-FVIII IgG, IdeS can restore the hemostatic efficacy of FVIII and provide a therapeutic option for inhibitor-positive patients.
Article
Biochemistry & Molecular Biology
Hsin-Tzu Liu, Yu-Ning Lin, Ming-Cheng Tsai, Ya-Chi Wu, Ming-Chung Lee
Summary: The study demonstrates that baicalein can attenuate LPS-induced depression-like behavior by suppressing neuroinflammation and inflammation induced by the peripheral immune response.
Article
Endocrinology & Metabolism
Kristen Skvorak, Joyce Liu, Nikki Kruse, Roasa Mehmood, Subhamoy Das, Stephan Jenne, Chinping Chng, U. Loi Lao, Da Duan, Jonathan Asfaha, Faye Du, Leann Teadt, Antionette Sero, Charlene Ching, James Riggins, Lianne Pope, Ping Yan, Harminder Mashiana, Moulay Hicham Alaoui Ismaili, Kerryn McCluskie, Gjalt Huisman, Adam P. Silverman
Summary: Maple syrup urine disease (MSUD) is a genetic disorder that affects branched-chain amino acid metabolism and can lead to severe neurotoxicity. Patients must follow a strict protein-restricted diet, but there are no effective therapeutic options available. In this study, researchers developed an oral enzyme therapy that can degrade leucine in the gastrointestinal tract, successfully reducing plasma leucine levels in animal models. This treatment has the potential to be a new therapy for MSUD patients.
JOURNAL OF INHERITED METABOLIC DISEASE
(2023)
Article
Biochemistry & Molecular Biology
Michael Bette, Eileen Cors, Carolin Kresse, Burkhard Schuetz
Summary: The study found that medical ozone has selective neuroprotective and possibly anti-inflammatory effects in SOD1(G93A) ALS mice, slowing down neurodegeneration, reducing SOD1-related vacuolization, and affecting microglial and astroglial responses.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2022)
Article
Biochemistry & Molecular Biology
L. Falese, K. Sandza, B. Yates, S. Triffault, S. Gangar, B. Long, L. Tsuruda, B. Carter, C. Vettermann, S. J. Zoog, S. Fong
Article
Pathology
Sima Z. Torabian, David de Semir, Mehdi Nosrati, Sepideh Bagheri, Altaf A. Dar, Sylvia Fong, Yong Liu, Scot Federman, Jeff Simko, Chris Haqq, Robert J. Debs, Mohammed Kashani-Sabet
AMERICAN JOURNAL OF PATHOLOGY
(2009)
Article
Cell Biology
Sylvia Fong, Frank King, Emma Shtivelman
Article
Oncology
Anne J. Zielinski, Sylvia Fong, Juanita Allison, Misako Kawahara, Jean-Philippe Coppe, Heidi Feiler, Nancy M. Lee, Pierre-Yves Desprez
INTERNATIONAL JOURNAL OF CANCER
(2010)
Article
Oncology
Ruth Chu, Xiaoyue Zhao, Chandi Griffin, Richard E. Staub, Mark Shoemaker, Joan Climent, Dale Leitman, Isaac Cohen, Emma Shtivelman, Sylvia Fong
INTERNATIONAL JOURNAL OF CANCER
(2010)
Article
Oncology
Jelena Klawitter, Jost Klawitter, Jennifer Gurshtein, Kyler Corby, Sylvia Fong, Mary Tagliaferri, Linda Quattrochi, Isaac Cohen, Emma Shtivelman, Uwe Christians
INTERNATIONAL JOURNAL OF CANCER
(2011)
Article
Multidisciplinary Sciences
Frank W. King, Sylvia Fong, Chandi Griffin, Mark Shoemaker, Rick Staub, Yan-Ling Zhang, Isaac Cohen, Emma Shtivelman
Article
Multidisciplinary Sciences
Vivian Chen, Richard E. Staub, Sylvia Fong, Mary Tagliaferri, Isaac Cohen, Emma Shtivelman
Article
Multidisciplinary Sciences
Horace DeLisser, Yong Liu, Pierre-Yves Desprez, Ann Thor, Paraskevei Briasouli, Chakrapong Handumrongkul, Jonathon Wilfong, Garret Yount, Mehdi Nosrati, Sylvia Fong, Emma Shtivelman, Melane Fehrenbach, Gaoyuan Cao, Dan H. Moore, Shruti Nyack, Denny Liggitt, Mohammed Kashani-Sabet, Robert Debs
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
(2010)
Article
Biochemistry & Molecular Biology
Joseph C. Chen, Amanda R. Luu, Nathan Wise, Rolando De Angelis, Vishal Agrawal, Linley Mangini, Jon Vincelette, Britta Handyside, Harry Sterling, Melanie J. Lo, Hio Wong, Nicole Galicia, Glenn Pacheco, Jeremy Van Vleet, Alexander Giaramita, Sylvia Fong, Sushmita M. Roy, Chuck Hague, Roger Lawrence, Sherry Bullens, Terri M. Christianson, Alessandra d'Azzo, Brett E. Crawford, Stuart Bunting, Jonathan H. LeBowitz, Gouri Yogalingam
JOURNAL OF BIOLOGICAL CHEMISTRY
(2020)
Article
Medicine, Research & Experimental
Lening Zhang, Britta Handyside, Ryan Murphy, Choong-Ryoul Sihn, Lin Xie, Catherine Vitelli, Danielle Harmon, Silvia Siso, Su Liu, Sherry Bullens, Stuart Bunting, Sylvia Fong
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT
(2020)
Article
Medicine, Research & Experimental
Sylvia Fong, Britta Handyside, Choong-Ryoul Sihn, Su Liu, Lening Zhang, Lin Xie, Ryan Murphy, Nicole Galicia, Bridget Yates, Wesley C. Minto, Catherine Vitelli, Danielle Harmon, Yuanbin Ru, Guoying Karen Yu, Claudia Escher, Jakob Vowinckel, Jill Woloszynek, Hassib Akeefe, Rajeev Mahimkar, Sherry Bullens, Stuart Bunting
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT
(2020)
Article
Biochemistry & Molecular Biology
Sylvia Fong, Bridget Yates, Choong-Ryoul Sihn, Aras N. Mattis, Nina Mitchell, Su Liu, Chris B. Russell, Benjamin Kim, Adebayo Lawal, Savita Rangarajan, Will Lester, Stuart Bunting, Glenn F. Pierce, K. John Pasi, Wing Yen Wong
Summary: Factor VIII gene transfer using valoctocogene roxaparvovec (AAV5-hFVIII-SQ) through a single intravenous infusion has shown clinical benefits lasting 5 years in patients with severe hemophilia A. This study investigated the molecular mechanisms underlying the sustained expression of AAV5-hFVIII-SQ-derived FVIII in the liver. The results demonstrated persistent episomal vector structures and provided insights into potential mechanisms mediating interindividual variability in transgene expression.
Article
Hematology
Sophia Stanford, Ruth Pink, Desmond Creagh, Amanda Clark, Gillian Lowe, Nicola Curry, John Pasi, David Perry, Sylvia Fong, Gregory Hayes, Kandiah Chandrakumaran, Savita Rangarajan
RESEARCH AND PRACTICE IN THROMBOSIS AND HAEMOSTASIS
(2019)