Review
Hematology
Mikhail V. Ovanesov, Joseph W. Jackson, Basil Golding, Timothy K. Lee
Summary: This article discusses the factors to consider when choosing an assay for potency assignment and postadministration monitoring of new factor products, including the validity of the assay calibrated with the IS, the meaning of potency values in IU, standards of care for patients, clinical relevance between the assigned potency value and recovery value from clinical laboratories, and patient safety.
JOURNAL OF THROMBOSIS AND HAEMOSTASIS
(2021)
Article
Immunology
Glaivy Batsuli, Jasmine Ito, Elizabeth S. York, Courtney Cox, Wallace Baldwin, Surinder Gill, Pete Lollar, Shannon L. Meeks
Summary: This study analyzed the internalization of FVIII complexed with epitope-mapped FVIII-specific IgG monoclonal antibodies by murine bone marrow-derived dendritic cells (BMDCs) in vitro, as well as the antibody development in hemophilia A (FVIII-/-) mice injected with FVIII-IC over time. The results showed that certain FVIII-IC subsets modulate the humoral response to FVIII in an epitope-dependent manner.
FRONTIERS IN IMMUNOLOGY
(2023)
Article
Hematology
Anne-Fleur Zwagemaker, Fabienne R. Kloosterman, Samantha C. Gouw, Sara Boyce, Paul Brons, Marjon H. Cnossen, Peter W. Collins, Jeroen Eikenboom, Charles Hay, Rutger C. C. Hengeveld, Shannon Jackson, Caroline A. M. Klopper-Tol, Marieke J. H. A. Kruip, Britta Laros-van Gorkom, Christoph Male, Laurens Nieuwenhuizen, Susan Shapiro, Karin Fijnvandraat, Michiel Copens
Summary: This study investigated the discrepancy between one-stage and chromogenic assays of coagulation factor activity in moderate and mild hemophilia A and B patients. The results showed that only a small number of patients exhibited a discrepancy between the two assays, and this discrepancy was largely determined by laboratory variables.
JOURNAL OF THROMBOSIS AND HAEMOSTASIS
(2023)
Article
Hematology
Viola J. F. Strijbis, Lorenzo G. R. Romano, Ka Lei Cheung, Jeroen Eikenboom, Ying Poi Liu, Andrew C. McCreary, Frank W. G. Leebeek, Mettine H. A. Bos
Summary: This study aimed to evaluate the efficacy of FIX-FIAV in plasma from HA patients. The results showed that FIX-FIAV could increase the FVIII-equivalent activity and coagulation activity in HA patient plasma, mitigating the HA phenotype. Therefore, FIX-FIAV could serve as a potential treatment for HA patients.
JOURNAL OF THROMBOSIS AND HAEMOSTASIS
(2023)
Article
Biochemistry & Molecular Biology
Junzheng Wu, Hang Zhang, Tong Lian, Yaling Ding, Chunlei Song, Dekuan Li, Liheng Wu, Tao Lei, Hong Liang
Summary: A novel B-domain-deleted recombinant FVIII was developed in this study without the use of animal or human serum-derived components. rFVIII promoted the generation of activated factor X and downstream thrombin, and exhibited ideal binding affinity to human von Willebrand factor. In vitro and in vivo experiments demonstrated the satisfactory efficacy and potency of rFVIII, suggesting its potential as an effective treatment strategy for FVIII deficiency.
BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS
(2023)
Article
Biology
Jeong Pil Han, Dong Woo Song, Jeong Hyeon Lee, Geon Seong Lee, Su Cheong Yeom
Summary: Innovative gene therapy is advancing towards functional restoration, with hemophilia being a representative genetic disorder. A novel mouse model of hemophilia A has been developed in this study, which could be utilized for future gene correction preclinical trials.
Article
Hematology
Amy Dericquebourg, Mathilde Fretigny, Nicolas Chatron, Brigitte Tardy, Christophe Zawadzki, Herve Chambost, Christine Vinciguerra, Yohann Jourdy
Summary: This study aimed to characterize deep intronic variants in hemophilia B patients for whom genetic investigations failed. Whole F9 sequencing was performed in 17 genetically unsolved hemophilia B patients. A total of 9 candidate variants were identified and their pathogenic impact was studied. Five variants were classified as likely pathogenic and four variants remained of unknown significance. The study successfully identified the hemophilia B-causing variant in 76% of the families included.
JOURNAL OF THROMBOSIS AND HAEMOSTASIS
(2023)
Article
Hematology
Paolo Simioni, Stefano Cagnin, Francesca Sartorello, Gabriele Sales, Luca Pagani, Cristiana Bulato, Sabrina Gavasso, Francesca Nuzzo, Francesco Chemello, Claudia M. Radu, Daniela Tormene, Luca Spiezia, Tilman M. Hackeng, Elena Campello, Elisabetta Castoldi
Summary: This study identified the molecular basis of high FVIII levels leading to severe thrombophilia in two Italian families, involving a tandem duplication of the proximal portion of the F8 gene. This rearrangement led to upregulation of F8 messenger RNA and transcriptional enhancement, potentially mediating the severe thrombophilia observed in carriers of the duplication.
Article
Cardiac & Cardiovascular Systems
Brandon Tse, Rosane Nisenbaum, Georgina Floros, Aziz Jiwajee, Jerome Teitel, Michelle Sholzberg
Summary: Continuous factor VIII (FVIII) or factor IX (FIX) infusions are commonly used for patients with hemophilia A (HA) or B (HB) undergoing surgery. This study found differences in initial recovery and subsequent FIX and FVIII levels, as well as clinical outcomes, among HB and HA patients receiving perioperative continuous infusion (CI) of recombinant FVIII and FIX concentrates. HB patients had higher initial bolus dose and longer length of hospital stay, suggesting a need for a higher CI rate for adequate surgical hemostatic coverage.
JOURNAL OF THROMBOSIS AND THROMBOLYSIS
(2023)
Article
Hematology
Nasim Shahidi Hamedani, Anouk Anna Marie Therese Donners, Matthijs van Luin, Simone Gasper, Heiko Ruehl, Claudia Klein, Thilo Albert, Mohsin EL Amrani, Bernd Poetzsch, Johannes Oldenburg, Jens Mueller
Summary: This study evaluated the impact of plasma FVIII activity on determining emicizumab levels and proposed different strategies for correcting or preanalytical inhibition of FVIII. The results showed that inhibitor-based FVIII neutralization is a feasible strategy for accurately measuring plasma emicizumab levels in the presence of FVIII activity.
JOURNAL OF THROMBOSIS AND HAEMOSTASIS
(2023)
Article
Hematology
Nhan H. Nguyen, Robert K. Dingman, Sathy V. Balu-Iyer
Summary: The study demonstrates that using FVIII associated with Lyso-PS nanoparticles significantly reduces inhibitor development and improves the plasma survival of FVIII following intravenous administration. Additionally, reduction in inhibitor formation can also be achieved using Lyso-PS nanoparticles through the user-friendly oral route of administration.
JOURNAL OF THROMBOSIS AND HAEMOSTASIS
(2021)
Article
Biochemistry & Molecular Biology
Jie Gong, Rui Yang, Min Zhou, Lung-Ji Chang
Summary: This study developed advanced lentiviral vectors for targeted expression of factor VIII (FVIII) and conducted functional experiments in human endothelial and megakaryocytic cell lines. In a mouse model, one of the vectors showed excellent packaging and delivery efficiencies, with endothelial specificity and low immunogenicity, suggesting its potential for clinical applications.
MOLECULAR MEDICINE
(2023)
Article
Hematology
Jacob Lund, Kasper Jensen, Laurent Burnier, Mirella Ezban
Summary: This study aimed to investigate the response of Mim8 in thrombin generation assays and its combination with other hemostatic proteins. The results showed that Mim8 has a similar mode of action with FVIII and the concomitant use of FVIII and Mim8 for bleed treatment is safe.
JOURNAL OF THROMBOSIS AND HAEMOSTASIS
(2023)
Article
Hematology
Amelia R. Wilhelm, Nicole A. Parsons, Benjamin J. Samelson-Jones, Robert J. Davidson, Charles T. Esmon, Rodney M. Camire, Lindsey A. George
Summary: This study compared an APC-resistant FVIII variant with wild-type FVIII, and found that APC plays an important role in the in vivo regulation of FVIIIa, which could be exploited for developing novel treatments for hemophilia A.
Article
Hematology
Laura H. Bukkems, Tim Preijers, Max W. F. van Spengler, Frank W. G. Leebeek, Marjon H. Cnossen, Ron A. A. Mathot
Summary: This study used in silico simulations to analyze the pharmacokinetic differences of various extended half-life factor VIII concentrates, with BAY 94-9027 showing the largest increase in AUC and best target attainment compared to standard half-life factor VIII.
THROMBOSIS AND HAEMOSTASIS
(2021)
Article
Agriculture, Dairy & Animal Science
Fernanda Borges Duarte, Marcelo de Macedo Brigido, Eduardo de Oliveira Melo, Sonia Nair Bao, Carlos Frederico Martins
Summary: Cells from different origins behave differently in terms of exogenous DNA transfection and formation of transgenic cells. Adipocytes show a better response to transfection regardless of the transfection reagent used. The presence of anti-CD3 was also detected in co-transfected cells.
ANIMAL BIOTECHNOLOGY
(2022)
Article
Biochemistry & Molecular Biology
Ronny Petterson dos Santos Araujo, Renato Kaylan Alves Franca, Napoleao Fonseca Valadares, Andrea Queiroz Maranhao, Marcelo Macedo Brigido
Summary: The study suggests that autoimmunity may have its origins in early repertoire selection in developmental B cells, shaping a primary repertoire by selecting B cells that can efficiently perform productive signaling. These B cells are stimulated by self-antigens such as DNA in the bone marrow, potentially leading to autoimmunity.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2021)
Article
Immunology
Natalia Gil-Jaramillo, Amanda Pereira Rocha, Taina Raiol, Flavia Nader Motta, Cecilia Favali, Marcelo M. Brigido, Izabela M. D. Bastos, Jaime M. Santana
Summary: The study identified viral infection response as the most regulated pathway during the interaction between T. cruzi and human DCs, and also found up-regulation of other immune responses. Additionally, increased gene expression of alternative inflammasome sensors during T. cruzi infection was identified for the first time.
FRONTIERS IN IMMUNOLOGY
(2021)
Review
Pharmacology & Pharmacy
Anderson dos Santos Ramos, Giovana Cristina Santana Viana, Marcelo de Macedo Brigido, Juliana Franco Almeida
Summary: Neutrophils, particularly their ability to form extracellular traps (NETs), play a significant role in the pathophysiology of inflammatory bowel disease (IBD). Studies have shown increased levels of NETs in tissue and serum samples from IBD patients, indicating their involvement in disease progression. This highlights the potential of targeting NETs as a therapeutic strategy to expand the current spectrum of IBD therapies.
PHARMACOLOGICAL RESEARCH
(2021)
Review
Biochemistry & Molecular Biology
Angela Donato Maia Malaquias, Livia Erika Carlos Marques, Soraya S. Pereira, Cleberson de Freitas Fernandes, Andrea Queiroz Maranhao, Rodrigo G. Stabeli, Eridan Orlando Pereira Tramontina Florean, Maria Izabel Florindo Guedes, Carla Freire Celedonio Fernandes
Summary: VHHs are small functional immunoglobulins that exhibit high tissue penetration and low immunogenicity. They provide superior antigen recognition compared to human antibodies, along with better solubility and stability. Plant-based expression systems offer versatility, cost-effectiveness, safety, and efficiency in producing VHH-related constructs.
INTERNATIONAL JOURNAL OF BIOLOGICAL MACROMOLECULES
(2021)
Article
Multidisciplinary Sciences
Ana Clara Barbosa Antonelli, Vinnycius Pereira Almeida, Fernanda Oliveira Feitosa de Castro, Jacyelle Medeiros Silva, Irmtraut Araci Hoffmann Pfrimer, Edecio Cunha-Neto, Andrea Queiroz Maranhao, Marcelo Macedo Brigido, Renato Oliveira Resende, Anamelia Lorenzetti Bocca, Simone Goncalves Fonseca
Summary: In this study, an in silico universal vaccine against Zika virus was developed, which is predicted to induce broad and high-coverage cellular and humoral immune responses.
SCIENTIFIC REPORTS
(2022)
Article
Biochemistry & Molecular Biology
Renato Kaylan Alves de Oliveira Franca, Jacyelle Medeiros Silva, Lucas Silva Rodrigues, Dimitri Sokolowskei, Marcelo Macedo Brigido, Andrea Queiroz Maranhao
Summary: This study utilized phage display technology to select human antibodies targeting the fusion loop region of the Zika virus. Three antibodies that can neutralize Zika virus infection were obtained through four rounds of selection. Due to the high conservation of the fusion loop region, these new antibodies have the potential to be used in therapeutic interventions against Zika virus and other flavivirus illnesses.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2022)
Article
Agriculture, Dairy & Animal Science
Concepta M. McManus, Carolina Madeira Lucci, Andrea Queiroz Maranhao, Daniel Pimentel, Felipe Pimentel, Samuel Rezende Paiva
Summary: This article reviews the impact of heat stress on sheep and goats, focusing on the physical, physiological, metabolic, genetic, and molecular pathways involved. The findings highlight the importance of molecular functions and biological processes in the response to heat stress.
Editorial Material
Agriculture, Dairy & Animal Science
Concepta McManus, Felipe Pimentel, Andre M. de Almeida, Daniel Pimentel
Summary: This paper presents a bibliographic mapping analysis of publications in the journal Tropical Animal Health and Production (TAHP) using data from Scopus. The analysis provides valuable insights for self-evaluation, scope assessment, and future direction of the journal. The findings indicate improvements in metrics such as Article Influence, Percentage of papers in Open Access, Immediacy Index, and Journal Impact Factor. However, further enhancements are still needed. The journal plays a crucial role in advancing knowledge of animal health and production in tropical and sub-tropical regions.
TROPICAL ANIMAL HEALTH AND PRODUCTION
(2023)
Review
Virology
Renato Kaylan Alves Franca, Igor Cabral Studart, Marcus Rafael Lobo Bezerra, Larissa Queiroz Pontes, Antonio Marcos Aires Barbosa, Marcelo Macedo Brigido, Gilvan Pessoa Furtado, Andrea Queiroz Maranhao
Summary: The search for innovative anti-cancer drugs remains challenging, but antibodies have proven to be a valuable asset in successful cancer therapy. The traditional hybridoma technology has limitations, necessitating alternative approaches like humanizing murine monoclonal antibodies. Phage Display libraries offer an innovative tool for antibody selection, allowing the in vitro selection of antibodies directed towards cancer antigens.
Article
Multidisciplinary Sciences
Concepta McManus, Abilio A. Baeta Neves, Jose Alexandre Diniz Filho, Andrea Q. Maranhao, Antonio G. Souza Filho
Summary: Brazilian science has shown steady improvement in quantity and quality over the past 20 years, but has recently faced financial restrictions since 2015. Increased international collaboration has led to higher citation impact, with diverse research areas showcasing international excellence.
ANAIS DA ACADEMIA BRASILEIRA DE CIENCIAS
(2021)