Results 61 to 70 of about 22,597,991 (289)

Perinatal Gene Transfer to the Liver [PDF]

, 2011
The liver acts as a host to many functions hence raising the possibility that any one may be compromised by a single gene defect. Inherited or de novo mutations in these genes may result in relatively mild diseases or be so devastating that death within
Buckley, SM, Chan, JK, Howe, SJ, McKay, TR, Rahim, AA, Waddington, SN, Ward, NJ   +6 more
core   +1 more source

Cross‐reacting inhibitors against recombinant porcine factor VIII in acquired hemophilia A: Data from the GTH‐AH 01/2010 Study

Journal of Thrombosis and Haemostasis, 2020
Recombinant porcine factor VIII (rpFVIII, OBI‐1, susoctocog alfa) is used for the treatment of acute bleeds in patients with acquired hemophilia A (AHA). Inhibitors in AHA can sometimes cross‐react with rpFVIII.
H. Türkantoz, C. Königs, P. Knöbl, R. Klamroth, K. Holstein, A. Huth‐Kühne, J. Heinz, H. Eichler, A. Tiede   +8 more
semanticscholar   +1 more source

Assessing the potential cost-effectiveness of a gene therapy for the treatment of hemophilia A

Journal of Medical Economics, 2020
Aim: Hemophilia A is a genetic, chronic disorder classified by deficient or defective coagulation factor VIII (FVIII) that puts those affected at risk for spontaneous bleeding episodes, which lead to joint damage and chronic pain over time.
Keziah Cook, Shaun P. Forbes, K. Adamski, Janice J Ma, A. Chawla, L. Garrison   +5 more
semanticscholar   +1 more source

Clinical, instrumental, serological and histological findings suggest that hemophilia B may be less severe than hemophilia A

Haematologica, 2016
Recent evidence suggests that patients with severe hemophilia B may have a less severe disease compared to severe hemophilia A. To investigate clinical, radiological, laboratory and histological differences in the arthropathy of severe hemophilia A and ...
Daniela Melchiorre, Silvia Linari, Mirko Manetti, Eloisa Romano, Francesco Sofi, Marco Matucci-Cerinic, Christian Carulli, Massimo Innocenti, Lidia Ibba-Manneschi, Giancarlo Castaman   +9 more
doaj   +1 more source

Recent Advances in mRNA Delivery Systems for Cancer Therapy

Advanced Science, EarlyView.
This review systematically investigates the applications of mRNA therapy in cancer treatment, with particular emphasis on nonviral delivery systems, targeting strategies, stimulus‐responsive systems, and local delivery methods. Concluding with a meticulous evaluation, the review sheds light on the prevailing challenges while illuminating promising ...
Zheng Zhang, Ya‐Nan Fan, Si‐Qi Jiang, Ya‐Jing Ma, Yao‐Ru Yu, Yu‐Xin Qing, Qian‐Ru Li, Yi‐Lin Liu, Song Shen, Jun Wang   +9 more
wiley   +1 more source

Hemophilia A and B: molecular and clinical similarities and differences

Haematologica, 2019
Several lines of evidence seem to suggest that the clinical features of hemophilia A and B patients may differ in several points. Genetic background shows a prevalence of missense mutations in hemophilia B compared to the high percentage of mutations ...
G. Castaman, Davide Matino
semanticscholar   +1 more source

Von Willebrand Disease [PDF]

, 2017
Von Willebrand Disease (VWD) is a blood clotting disorder characterized by low levels of the Von Willebrand Factor (VWF) in the blood. VWF is functions to bind platelets and promote clotting of the blood when vascular injury occurs.
Misla David, Ramon
core   +2 more sources

Standardization of Terminology, Definitions, and Outcome Criteria for Bleeding in Hereditary Hemorrhagic Telangiectasia: International Consensus Report

American Journal of Hematology, EarlyView.
ABSTRACT Hereditary hemorrhagic telangiectasia (HHT, Osler‐Weber‐Rendu disease) is the second most common inherited bleeding disorder worldwide, affecting approximately 1 in 5000 people. Development of disease‐modifying and efficacious hemostatic agents to treat HHT has finally begun after decades without such medical therapies.
Hanny Al‐Samkari, Raj S. Kasthuri, Hans‐Jurgen Mager, Jenny Y. Zhou, Marcelo M. Serra, Bethany T. Samuelson‐Bannow, Layla N. Van Doren, Jay F. Piccirillo, Marianne S. Clancy, Keith R. McCrae, Sonia M. Thomas, Antoni Riera‐Mestre, Allyson M. Pishko, Sarah Sewaralthahab, James R. Gossage, Vivek N. Iyer, Cedric Hermans, Adrienne Hammill, Ingrid Winship, Meir Mei‐Zahav, Annette von Drygalski, Scott Olitsky, Marie E. Faughnan   +22 more
wiley   +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

Advances on Albumin‐Based Carriers for Anticancer Drug Delivery

Advanced NanoBiomed Research, EarlyView.
This study systematically classifies the drug delivery modes of albumin by examining the characteristics of its surface‐active groups and internal binding sites. It provides an in‐depth analysis of various drug delivery strategies and their corresponding targeting mechanisms.
Ruoli Zhou, Rongbin Zhong, Zhonghui Luo, Hua Wei, Cui‐Yun Yu   +4 more
wiley   +1 more source