Results 131 to 140 of about 22,562,840 (346)

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

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

Adherence to prophylaxis in adolescents and young adults with severe haemophilia: A quantitative study with patients [PDF]

, 2017
© 2017 van Os et al. This is an open access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are ...
Hart, Daniel, Sullivan, Keith, Troop, Nicholas, Van Os, Sandra   +3 more
core   +5 more sources

THE MUTATION RATE OF THE GENE FOR HAEMOPHILIA, AND ITS SEGREGATION RATIOS IN MALES AND FEMALES [PDF]

, 1946
J. B. S. Haldane
openalex   +1 more source

Measuring activities and participation in persons with haemophilia: A systematic review of commonly used instruments

Haemophilia, 2018
Monitoring clinical outcome in persons with haemophilia (PWH) is essential in order to provide optimal treatment for individual patients and compare effectiveness of treatment strategies.
M. Timmer, S. Gouw, B. Feldman, A. Zwagemaker, P. Kleijn, M. Pisters, M. Pisters, R. Schutgens, V. Blanchette, A. Srivastava, J. David, K. Fischer, J. Net   +12 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

A new paradigm evaluating cost per cure of HCV infection in the UK [PDF]

, 2016
Background: New interferon (IFN)-free treatments for hepatitis C are more effective, safer but more expensive than current IFN-based therapies. Comparative data of these, versus current first generation protease inhibitors (PI) with regard to costs and ...
Aline Gauthier, Elizabeth Holtham, Graham S. Cooke, Jeremie Schwarzbard, null null, Petar Atanasov, Stephen T. Barclay, William L. Irving   +7 more
core   +2 more sources

Immune Tolerance Induction With a Recombinant Factor VIII Fc in Haemophilia A: Data From a Chart Review Study. [PDF]

Eur J Haematol
ABSTRACT Objective To report data from an ITI chart review study (NCT03951103) for first‐time and rescue ITI with recombinant factor VIII Fc fusion protein (rFVIIIFc) in persons with haemophilia A. Methods Retrospective and prospective real‐world data are reported from a non‐interventional, multicentre study of patients who had been or were currently ...
Klamroth R, Al Saleh M, Glosli H, Schiavulli M, Guillet B, Bystrická L, Schönstein A, Lethagen S.   +7 more
europepmc   +2 more sources

HAEMOSTATIC THERAPY IN HAEMOPHILIA [PDF]

, 1929
W. W. Payne, Riana Steen
openalex   +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