Results 61 to 70 of about 48,006 (264)

Gene Therapy and Gene Editing in Type 1 Diabetes: CRISPR‐Based β‐Cell Replacement and Treg Immune Modulation Approaches

Diabetes, Obesity and Metabolism, EarlyView.
ABSTRACT Background Type 1 diabetes mellitus (T1DM) is a chronic autoimmune disease marked by the destruction of pancreatic β‐cells, resulting in lifelong dependence on exogenous insulin. Despite advances in insulin delivery and glucose monitoring technologies, patients remain at risk for acute and long‐term complications, underscoring the need for ...
Tzu‐Min Lin, Hui‐Fang Chang, Tzu‐Ching Lin, Cheng‐Han Lin, Yu‐Ling Sun, Chih‐Sheng Lin   +5 more
wiley   +1 more source

POLYMORPHISM OF THROMBOPHILIA GENES AND THEIR ROLE IN DEVELOPMENT OF DIFFERENT DISEASE PHENOTYPES AND THROMBOTIC COMPLICATIONS IN HEMOPHILIA PATIENTS

Сибирский научный медицинский журнал, 2019
Objective was to study genetic markers of thrombophilia in patients with hemophilia, which can affect the course of the disease and contribute to thrombotic complications. Material and methods.
T. Yu. Polyanskaya, I. S. Fevraleva, E. E. Karpov, N. V. Sadykova, M. S. Sampiev, A. V. Golobokov, G. V. Mishin, D. Yu. Petrovskiy, A. A. Koroleva, G. M. Galstyan, A. B. Sudarikov, V. Yu. Zorenko   +11 more
doaj   +1 more source

Contrasting Approaches in the Implementation of GRADE Methodology in Guidelines for Haemophilia and Von Willebrand Disease

Haemophilia, EarlyView.
ABSTRACT Introduction The 2024 ISTH clinical practice guideline (CPG) for treatment of congenital haemophilia, the NBDF‐McMaster Guideline on Care Models for Haemophilia Management, and ASH ISTH NBDF WFH guidelines on the diagnosis and management of VWD all utilised GRADE methodology.
Mark W. Skinner, Manuela Albisetti, Jesús Ardila, Jan Astermark, Jan Blatny, Manuel Carcao, Pratima Chowdary, Nathan T. Connell, Miguel Crato, Yesim Dargaud, Roseline d'Oiron, Amy L. Dunn, Miguel A. Escobar, Carmen Escuriola‐Ettingshausen, Emna Gouider, Annie Harroche, Cedric Hermans, Victor Jimenez‐Yuste, Radoslaw Kaczmarek, Gili Kenet, Liane Khoo, Robert Klamroth, Florian Langer, David Lillicrap, Johnny Mahlangu, Christoph Male, Tadashi Matsushita, Sandrine Meunier, Wolfgang Miesbach, Beatrice Nolan, Johannes Oldenburg, Brian O'Mahony, Margareth Ozelo, Glenn F. Pierce, Gloria Ramos, Michael Recht, Olivia Romero‐Lux, Dawn Rotellini, Rita C. Santoro, Tammuella C. Singleton, Alok Srivastava, Sophie Susen, Kate Talks, Huyen Tran, Leonard A. Valentino, Jerzy Windyga, Renchi Yang, Maria Elisa Mancuso, also on behalf of AICE, AHAD‐AP, AHCDC, ATHN, CLAHT, EHC, French CRH & MHEMO, HTCCNC, NBDF, PEDNET, UKHCDO, WFH   +59 more
wiley   +1 more source

Could PTH/Ca Ratio Serve as a New Marker for Evaluating Bone Metabolism in Hemophilia Patients?

Diagnostics
Background/Objectives: Low bone mineral density (BMD) is common in hemophilia patients. Identifying high-risk patients for low BMD early is essential to prevent complications and reduce morbidity.
Tuba Ersal, Fazıl Çağrı Hunutlu, Vildan Gürsoy, Ezel Elgün, Şeyma Yavuz, İpek Dal Akkuş, İlayda Baş, Vildan Özkocaman, Fahir Özkalemkaş   +8 more
doaj   +1 more source

Human parvovirus B19 and blood product safety. A tale of twenty years of improvements [PDF]

, 2015
Parvovirus B19 (B19V), long known to be the causative agent of erythema infectiosum (fifth disease), is not a newly emerging agent. The aim of this review is to analyse the role played by this virus in compromising safety in transfusion medicine and the ...
Calizzani, Gabriele, Candura, Fabio, Facco, Giuseppina, Grazzini, Giuliano, Liumbruno, Giancarlo M., Marano, Giuseppe, Pupella, Simonetta, VAGLIO, Stefania   +7 more
core   +1 more source

Central Nervous System Bleeding in Children With Haemophilia in Limited Resource

Haemophilia, EarlyView.
ABSTRACT Introduction Central nervous system (CNS) bleeding in children with haemophilia is a life‐threatening complication that may cause severe neurological sequelae or death. In resource‐limited settings, where prophylaxis is not universally accessible, its patterns and outcomes may differ from those in high‐income countries.
Patcharee Komvilaisak, Sudarat Sirichaipornsak, Paiporn Sripraya, Napat Laoaroon, Kunanya Suwannaying, Pichayen Duanthongphon   +5 more
wiley   +1 more source

PRODUCTION OF HIGH LEVELS OF TRANSGENC FACTOR IX WITHOUT GENE RESCUE, AND ITS THERAPEUTIC USES [PDF]

, 2011
A non-human transgenic mammalian animal, as described above, contains an exogenous double stranded DNA sequence stably integrated into the genome of the animal, which comprises cis-acting regulatory units operably linked to a DNA sequence encoding human ...
Cooper, Julian D., O\u27Sickey, Tanya K., Velander, William Hugold   +2 more
core   +1 more source

Enhancing the Evidence for Care in Underserved Bleeding Disorders Communities

Haemophilia, EarlyView.
ABSTRACT Background Major advances in haemophilia care have not translated equitably across all populations. Individuals with rare bleeding disorders (RBDs), people living in low‐ and lower‐middle‐income countries (LMICs) and women and girls with inherited bleeding disorders (WGWBD) continue to face significant diagnostic, therapeutic and research ...
Johnny Mahlangu
wiley   +1 more source

Christmas Disease (Hemophilia –B) – A Case Report

Bangabandhu Sheikh Mujib Medical University Journal, 2010
We report a 25 years old man developed Haemarthrosis of left hip joint with a history of recurrent swelling and pain in multiple joints and prolonged bleeding following minor trauma since childhood.
Md Rafiqul Alam, Mohammad Saiful Habib, Mohammad Arifur Rahman, Md Mizanur Rahman Khan, MA Jalil Chowdhury, Taimur AK Mahmud   +5 more
doaj   +1 more source

A viable mouse model of factor X deficiency provides evidence for maternal transfer of factor X. [PDF]

, 2008
BackgroundActivated factor X (FXa) is a vitamin K-dependent serine protease that plays a pivotal role in blood coagulation by converting prothrombin to thrombin. There are no reports of humans with complete deficiency of FX, and knockout of murine F10 is
Arruda, VR, Chu, K, Golden, JA, Herzog, RW, High, KA, Labosky, PA, Margaritis, P, Tai, SJ   +7 more
core   +2 more sources