Results 21 to 30 of about 50,045 (313)

Cochlear implantation in hemophilia B—a rare case report

The Egyptian Journal of Otolaryngology, 2022
Background Hemophilia B is an X-linked inherited disease, mainly caused by deficiency of factor IX. Severity of the disease is manifested by the factor IX deficiency in the blood.
Chetan Yadav, Diva Sharma, Neeraj Suri, Meenesh Juvekar   +3 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

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

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

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

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

Haemophilia A: pharmacoeconomic review of prophylaxis treatment versus on-demand [PDF]

, 2015
Objectives. Haemophilia A is a congenital disorder of coagulation that mainly affects males and causes a considerable use of resources, especially when hemophilic patients are treated with prophylaxis.
BOCCIA, Antonio, LA TORRE, Giuseppe, Ricciardi, Walter, UNIM, BRIGID ANDOUNIMYE, Veneziano, Maria Assunta   +4 more
core   +2 more sources

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