Results 91 to 100 of about 69,645 (303)

International Perspectives on Vitamin K Deficiency Bleeding in Infants: A Cross‐Sectional Questionnaire‐Based Survey

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Purpose Vitamin K deficiency bleeding (VKDB) can occur in neonates and infants due to low placental transfer and storage of VK. Although the incidence has decreased with global VK prophylaxis at birth, VKDB remains a concern. This study aimed to assess the recent magnitude, risk factors, and outcomes of VKDB and barriers/limitations to VK ...
Nongnuch Sirachainan, Patcharee Komvilaisak, C. Heleen van Ommen, Shoshana Revel‐Vilk, Shouichi Ohga, Ayesha Zia, Masayuki Ochiai, Daijiro Takahashi, Shutaro Suga, Veerle Labarque, Ernest K. Amankwah, Bounpalisone Souvanlasy, Marisol Betensky, Anthony KC Chan, Ampaiwan Chuansumrit, Neil Goldenberg, Madhvi Rajpurkar   +16 more
wiley   +1 more source

Hemophilia A: An Ideal Disease for Prenatal Therapy

Prenatal Diagnosis, EarlyView.
ABSTRACT Hemophilia A (HA) is the most common inherited coagulation defect. Current state‐of‐the‐art treatment consists of frequent administration of prophylactic infusions of coagulation factor VIII (FVIII) protein or bispecific antibodies that replace the cofactor function of FVIIIa to maintain hemostasis. However, these treatments are far from ideal,
Christopher D. Porada, Anthony Atala, Graça Almeida‐Porada   +2 more
wiley   +1 more source

Novel variant in HPS3 gene in a patient with Hermansky Pudlak syndrome (HPS) type 3

Platelets, 2020
Hermansky-Pudlak syndrome (HPS) is a rare autosomal recessive disorder caused by defects in 10 human HPS genes, characterized by oculocutaneous albinism (OCA) and bleeding diathesis associated to platelet δ-storage pool defect (SPD).
Anna Lecchi, Silvia La Marca, Eti A Femia, Antonia Lenz, Doris Boeckelmann, Andrea Artoni, Flora Peyvandi, Barbara Zieger   +7 more
doaj   +1 more source

Nature Inspired Delivery Vehicles for CRISPR‐Based Genome Editing

Small, EarlyView.
The review highlights nature‐inspired nanocarriers for CRISPR delivery, emphasizing viral vectors, extracellular vesicles, liposomes, and lipid nanoparticles. It discusses their roles in improving specificity, minimizing immunogenicity, and overcoming barriers in genome editing. Recent advancements, challenges, and therapeutic applications are explored,
Elizabeth Maria Clarissa, Mamata Karmacharya, Hyunmin Choi, Sumit Kumar, Yoon‐Kyoung Cho   +4 more
wiley   +1 more source

Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia.

New England Journal of Medicine, 2007
BACKGROUND Effective ways to prevent arthropathy in severe hemophilia are unknown. METHODS We randomly assigned young boys with severe hemophilia A to regular infusions of recombinant factor VIII (prophylaxis) or to an enhanced episodic infusion ...
M. Manco‐Johnson, Thomas C Abshire, A. Shapiro, B. Riske, Michele R. Hacker, R. Kilcoyne, J. D. Ingram, Michael L. Manco-Johnson, S. Funk, L. Jacobson, Leonard A. Valentino, W. Hoots, George R. Buchanan, D. DiMichele, M. Recht, Deborah L Brown, C. Leissinger, Shirley Bleak, Alan R. Cohen, P. Mathew, A. Matsunaga, D. Medeiros, D. Nugent, Gregory A. Thomas, Alexis A. Thompson, K. McRedmond, J. M. Soucie, H. Austin, Bruce L. Evatt   +28 more
semanticscholar   +1 more source

Dynamic Liposome Sensing Platform to Wirelessly Ensure Nucleic Acid Encapsulation via Non‐Contact Perception

Small, EarlyView.
Inspired by the Ampullae of Lorenzini in elasmobranch fish, a biomimetic electroreceptor platform is developed using an electret layer‐coated single‐walled carbon nanotube thin‐film transistor (eSWCNT‐TFT). This platform enables non‐contact, label‐free, and real‐time monitoring of DNA encapsulation efficiency in liposome droplets by detecting net ...
Younsu Jung, Jinhwa Park, Seonghun Shin, Sunghee Kim, Bijendra Bishow Maskey, Kiran Shrestha, Jianfu Ding, Thi Thuy Vy Tran, Patrick R. L. Malenfant, Jinkee Lee, Jong‐Sun Kang, Gyoujin Cho   +11 more
wiley   +1 more source

GENETIC DIAGNOSTICS OF HEMOPHILIA A

Zdravniški Vestnik, 2004
Background. Hemophilia A is a chromosome X-linked bleeding disorder due to mutations in the FVIII gene. There are 163 hemophilia A patients in Slovene registry for Hemophilia.
Maruša Debeljak, Majda Benedik Dolničar, Lana Strmecki   +2 more
doaj  

Subcutaneous concizumab prophylaxis in hemophilia A and hemophilia A/B with inhibitors: Phase 2 trial results.

Blood, 2019
Results from the main parts (24 weeks) of two concizumab phase 2 trials are presented: explorer4 (NCT03196284) in hemophilia A (HA) or B (HB) with inhibitors (HAwI/HBwI); explorer5 (NCT03196297) in HA without inhibitors.
A. Shapiro, P. Angchaisuksiri, J. Astermark, G. Benson, G. Castaman, P. Chowdary, H. Eichler, V. Jiménez‐Yuste, K. Kavaklı, T. Matsushita, L. Poulsen, Allison P. Wheeler, G. Young, S. Zupancic-Salek, J. Oldenburg   +14 more
semanticscholar   +1 more source

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

Effectiveness of Octocog Alfa (BAY 81‐8973) to Treat People With Haemophilia A Enrolled in the ATHNdataset, Including Under‐Represented Subgroups

European Journal of Haematology, EarlyView.
ABSTRACT Objectives To evaluate the real‐world effectiveness of octocog alfa (BAY 81‐8973; Kovaltry) in people with haemophilia A (PwHA), including under‐represented subgroups. Methods We retrospectively analysed the American Thrombosis and Hemostasis Network (ATHN) dataset to identify the characteristics, treatment/clinical histories and annualised ...
Martin Chandler, Thomas Moulton, Lena Charafi, Jessica Charlet, Michael Recht   +4 more
wiley   +1 more source