Results 21 to 30 of about 48,006 (264)

A Case of Hemophilia B with Abdominal and Pelvic Pseudotumor

罕见病研究, 2022
Hemophilia B is a genetic disorder caused by coagulation factor Ⅸ(FⅨ) deficiency, mainly manifesting as joint, muscle and deep tissue bleeding. Hemophilia pseudotumor is a mass formed by soft tissue liquefaction and necrosis caused by repeated bleeding ...
ZHANG Jing, JU Mankai, ZHANG Kemin, CHEN Lingling, WANG Yuhua, ZHANG Lei, YANG Renchi, XUE Feng   +7 more
doaj   +1 more source

Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA. [PDF]

, 2001
Long-term expression of coagulation factor IX (FIX) has been observed in murine and canine models following administration of recombinant adeno-associated viral (rAAV) vectors into either the portal vein or muscle. These studies were designed to evaluate
Davidoff, A, Hanawa, H, Nathwani, AC, Nienhuis, AW, Vanin, EF, Zhou, JF   +5 more
core   +1 more source

Neonatal Gene Therapy for Hemophilia B by a Novel Adenovirus Vector Showing Reduced Leaky Expression of Viral Genes

Molecular Therapy: Methods & Clinical Development, 2017
Gene therapy during neonatal and infant stages is a promising approach for hemophilia B, a congenital disorder caused by deficiency of blood coagulation factor IX (FIX).
Shunsuke Iizuka, Fuminori Sakurai, Masashi Tachibana, Kazuo Ohashi, Hiroyuki Mizuguchi   +4 more
doaj   +1 more source

The history and evolution of the clinical effectiveness of haemophilia type a treatment: a systematic review. [PDF]

, 2012
First evidence of cases of haemophilia dates from ancient Egypt, but it was when Queen Victoria from England in the 19th century transmitted this illness to her descendants, when it became known as the "royal disease".
A Aleem, A Farrugia, A Miners, A Mocroft, AL Dunn, BA Konkle, BL Evatt, BM Feldman, CA Lee, CL Poel Van der, Claudia P. Casas, D Dimichele, D Dimichele, E Berntorp, E Berntorp, E Santagostino, Hector E. Castro, J Astermark, J Goudemand, J Oldenburg, J Stachnik, J Tusell, J Wight, Juan David Rueda, K Porter, L Valentino, L Valentino, LM Aledort, M Carcao, M Carcao, M Depka Von, M Franchini, M Lloyd, M Makris, María Fernanda Briceño, MD Carcao, MV Ragni, P Petrini, PM Mannucci, PM Mannucci, R Prabhu, S Royal, SA Larsson, SC Darby, SC Gouw, T Wong, TL Chorba, VS Blanchette, WK Hoots   +48 more
core   +2 more sources

Spontaneous bleeding in a patient with malignant lymphoma: A case of acquired hemophilia [PDF]

, 2000
Background: Acquired hemophilia is a rare condition which can be associated with lymphoproliferative disease. Case Report: Eleven yea rs after the diagnosis of immunocytoma had been made, a 72-year-old man developed a high-titer factor VIII inhibitor. At
Brack, N., Geiersberger, U., Gesierich, Wolfgang, Hartenstein, R., Munker, Reinhold, Pohlmann, H.   +5 more
core   +1 more source

Endothelial cells derived from patients' induced pluripotent stem cells for sustained factor VIII delivery and the treatment of hemophilia A. [PDF]

, 2020
Hemophilia A (HA) is a bleeding disorder characterized by spontaneous and prolonged hemorrhage. The disease is caused by mutations in the coagulation factor 8 gene (F8) leading to factor VIII (FVIII) deficiency.
Agu, Emmanuel, Conroy, Kelsey, Cortez-Toledo, Elizabeth, Gao, Kewa, Hyllen, Alicia A, Nolta, Jan A, Pan, Guangjin, Rose, Melanie, Wang, Aijun, Zhou, Ping   +9 more
core   +1 more source

Haemophilia B: an illustrative review of current challenges and opportunities

Research and Practice in Thrombosis and Haemostasis
Background: Hemophilia B is a genetic bleeding disorder caused by a deficiency of clotting factor IX, which presents unique challenges in clinical management.
Cedric Hermans, Jan Astermark, Sonata Šaulytė Trakymienė, Víctor Jiménez-Yuste   +3 more
doaj   +1 more source

Autologous and Heterologous Cell Therapy for Hemophilia B toward Functional Restoration of Factor IX

Cell Reports, 2018
Summary: Hemophilia B is an ideal target for gene- and cell-based therapies because of its monogenic nature and broad therapeutic index. Here, we demonstrate the use of cell therapy as a potential long-term cure for hemophilia B in our FIX-deficient ...
Suvasini Ramaswamy, Nina Tonnu, Tushar Menon, Benjamin M. Lewis, Kevin T. Green, Derek Wampler, Paul E. Monahan, Inder M. Verma   +7 more
doaj   +1 more source

Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway. [PDF]

, 2003
A systematic evaluation of the influence of sex on transduction by recombinant adeno-associated viral vector (rAAV) indicated that transgene expression after liver-targeted delivery of vector particles was between 5- to 13-fold higher in male mice ...
Davidoff, AM, Nathwani, AC, Ng, CYC, Spence, Y, Zhou, J   +4 more
core   +1 more source

Spontaneous Iliopsoas Hematoma following Microvascular Free Tissue Transfer. [PDF]

, 2017
Spontaneous hematoma within the iliopsoas muscle (SIH) is a rare complication most commonly seen in coagulopathic patients. Often, patients undergoing microvascular free tissue transfer are anticoagulated for anastomotic patency.
Alemi, A Sean, Faden, Daniel L, Heaton, Chase M, Markey, Jeffrey D, Naunheim, Margaret L, Seth, Rahul   +5 more
core   +2 more sources