Results 101 to 110 of about 13,086,034 (342)

Lipid Nanoparticles for Delivery of CRISPR Gene Editing Components

Small Methods, EarlyView.
The review presents a comprehensive overview of each component of lipid nanoparticles（LNPs）and their effects on editing efficiency. It specifically highlights strategies for achieving non‐liver delivery, aiming for broader applications in gene editing. Furthermore, this review summarizes the applications of LNPs in gene editing and offers insights for ...
Fan Wu, Nei Li, Yudian Xiao, Rohan Palanki, Hannah Yamagata, Michael J. Mitchell, Xuexiang Han   +6 more
wiley   +1 more source

Difficulties in the treatment of an Infant with Hemophilia B [PDF]

Türk Pediatri Arşivi, 2016
Dear Editor, Hemophilia B is a X-linked recessive bleeding disorder which occurs as a result of Factor IX (FIX) deficiency (1, 2). In some patients with hemophilia, antibody (inhibitor) and allergic reaction may develop against FIX protein which is administered for treatment (3).
Zulfikar, Bulent, Ozdemir, Gul Nihal, Dereci, Selim, Ozkasap, Serdar   +3 more
openaire   +4 more sources

Elucidating the etiology of idiopathic spontaneous intraperitoneal hemorrhage

Journal of Forensic Sciences, EarlyView.
Abstract Free blood within the abdominal cavity (hemoperitoneum) presents a significant diagnostic and interpretive challenge. It may result from trauma or occur spontaneously in association with underlying disease conditions. When no source of fatal hemorrhage is identified, the implications extend across forensic, criminalistic, legal, and ethical ...
Dalibor Kovařík, Štěpánka Pohlová Kučerová, Lenka Zátopková, Petr Hejna, Martin Janík   +4 more
wiley   +1 more source

Impact of hemophilia B on quality of life in affected men, women, and caregivers—Assessment of patient‐reported outcomes in the B‐HERO‐S study

European Journal of Haematology, 2018
Health‐related quality of life (HRQoL) is impaired in patients with hemophilia; however, the impact in mild/moderate hemophilia B and affected women is not well characterized.
T. Buckner, M. Witkop, C. Guelcher, R. Sidonio, C. Kessler, David B Clark, Wendy E. Owens, N. Frick, N. Iyer, D. Cooper   +9 more
semanticscholar   +1 more source

Outcomes and Healthcare Resource Utilisation in Adults With von Willebrand Disease Receiving On‐Demand Recombinant von Willebrand Factor in the United Kingdom

European Journal of Haematology, EarlyView.
ABSTRACT Objectives We describe treatment outcomes and healthcare resource utilisation (HCRU) in adults with von Willebrand disease (VWD) treated on demand with recombinant von Willebrand factor (rVWF) in the United Kingdom. Methods Retrospective chart review of adults (≥ 18 years) with congenital VWD receiving first‐time rVWF for the on‐demand ...
Mike Laffan, Heena Howitt, Cheryl Jones, Sarah Brighton, Rosa Willock, Anna Sanigorska, Oliver Heard   +6 more
wiley   +1 more source

Spectrum of hemophilia in Diyala-Iraq

Diyala Journal of Medicine, 2019
Background: Deficiencies of coagulation factors have been recognized for centuries. The most common inherited factor deficiencies are the hemophilias, X-linked diseases caused by deficiency of factor VIII (hemophilia A) or factor IX ( hemophilia B ...
Imad Ahmed Lateef, Hayder Jassim Hamood, Hayder Jassim Hamood, Oday Abbood Khaleel   +3 more
doaj   +2 more sources

Patient and parent preferences for characteristics of prophylactic treatment in hemophilia

Patient Preference and Adherence, 2015
Roberto Furlan,1 Sangeeta Krishnan,2 Jeffrey Vietri3 1Advanced Methods, Kantar Health, Epsom, Surrey, UK; 2Global Health Economics and Outcomes Research, Biogen, MA, USA; 3Health Outcomes, Kantar Health, Milan, Italy Introduction: New longer-acting ...
Furlan R, Krishnan S, Vietri J
doaj  

Anti-CD20 as the B-Cell Targeting Agent in a Combined Therapy to Modulate Anti-Factor VIII Immune Responses in Hemophilia a Inhibitor Mice [PDF]

, 2014
Neutralizing antibody formation against transgene products can represent a major complication following gene therapy with treatment of genetic diseases, such as hemophilia A.
Carol H. Miao, Chao Lien Liu, Chérie L. Butts, Jacqueline Lin, Peiqing Ye   +4 more
core   +1 more source

Dysfunctional endogenous FIX impairs prophylaxis in a mouse hemophilia B model.

Blood, 2019
Factor IX (FIX) binds to collagen IV (Col4) in the subendothelial basement membrane. In hemophilia B, this FIX-Col4 interaction reduces the plasma recovery of infused FIX and plays a role in hemostasis.
B. Cooley, G. Broze, D. Mann, F. Lin, L. Pedersen, D. Stafford   +5 more
semanticscholar   +1 more source

Outcomes and Healthcare Resource Utilisation in Adults With von Willebrand Disease Treated With Recombinant von Willebrand Factor in Surgical Settings in the United Kingdom

European Journal of Haematology, EarlyView.
ABSTRACT Objectives We describe treatment outcomes and healthcare resource utilisation (HCRU) in adults with von Willebrand disease (VWD) receiving recombinant von Willebrand factor (rVWF) in surgical settings in the United Kingdom. Methods Retrospective chart review of adults (≥ 18 years) with congenital VWD receiving first‐time rVWF for the ...
Mike Laffan, Heena Howitt, Cheryl Jones, Sarah Brighton, Rosa Willock, Anna Sanigorska, Oliver Heard   +6 more
wiley   +1 more source