Results 71 to 80 of about 69,645 (303)

Exploring the Potential and Advancements of Circular RNA Therapeutics

Exploration, EarlyView.
Given the remarkable advantages in terms of stability, sustained expression profile, safety, wide range of druggable targets, scalable and cost‐effective manufacturing capabilities, circRNA is currently undergoing intensive investigation for various therapeutic applications such as vaccines, protein replacement, genetic disease treatment, gene therapy,
Lei Wang, Lianqing Wang, Chunbo Dong, Jialong Liu, Guoxin Cui, Shan Gao, Zhida Liu   +6 more
wiley   +1 more source

Hemophilia treatment and transfusion medicine

Zhongguo shuxue zazhi
The improvement of hemophilia treatment is attributed to the development of transfusion medicine, and the discovery of cryoprecipitate has opened up a new era of hemophilia treatment, laying the foundation for the development of modern hemophilia ...
FANG Yunhai, CHENG Yan, ZHANG Xinsheng
doaj   +1 more source

Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.

New England Journal of Medicine, 2011
BACKGROUND Hemophilia B, an X-linked disorder, is ideally suited for gene therapy. We investigated the use of a new gene therapy in patients with the disorder.
A. Nathwani, E. Tuddenham, S. Rangarajan, Cecilia Rosales, J. McIntosh, D. Linch, P. Chowdary, A. Riddell, Arnulfo Pie, C. Harrington, J. O’Beirne, Keith A. Smith, J. Pasi, B. Glader, P. Rustagi, C. Ng, M. Kay, Junfang Zhou, Yunyu Spence, C. Morton, J. Allay, J. Coleman, S. Sleep, J. Cunningham, D. Srivastava, E. Basner-Tschakarjan, F. Mingozzi, K. High, J. Gray, U. Reiss, A. Nienhuis, A. Davidoff   +31 more
semanticscholar   +1 more source

Clinical, humanistic, and economic burden of severe hemophilia B in the United States: Results from the CHESS US and CHESS US+ population surveys

Orphanet Journal of Rare Diseases, 2021
Background Hemophilia B is a rare congenital bleeding disorder that has a significant negative impact on patients’ functionality and health-related quality of life.
T. Burke, S. Asghar, J. O’Hara, E. Sawyer, Nanxin Li   +4 more
semanticscholar   +1 more source

Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B.

Blood, 2018
Gene therapy for hemophilia B aims to ameliorate bleeding risk and provide endogenous factor IX (FIX) activity/synthesis through a single treatment, eliminating the requirement for FIX concentrate.
Wolfgang A. Miesbach, K. Meijer, M. Coppens, P. Kampmann, R. Klamroth, R. Schutgens, M. Tangelder, G. Castaman, J. Schwäble, H. Bonig, E. Seifried, F. Cattaneo, C. Meyer, F. Leebeek   +13 more
semanticscholar   +1 more source

Mobile Health Technology for the Personalized Therapy of Hemophilia

Frontiers in Medicine, 2021
The management of patients with hemophilia has evolved significantly since the first treatment attempts were made in the late 1930s. Since then, each new step in the treatment of patients with hemophilia has brought important advancements, as well as its
Noemi Dirzu, Ionut Hotea, C. Jitaru, Melen Brînză, L. Urian, Mareike-Catrina Peters, Krisztina Gál, Louis Popescu, C. Blag, M. Marian, É. Pál, M. Stanescu, Diana Cenariu, C. Tarniceriu, M. Serban, D. Dima, D. Coriu, C. Tomuleasa   +17 more
semanticscholar   +1 more source

Investigating the role of SARM1 in central nervous system

Ibrain, EarlyView.
Sterile‐α and Toll/interleukin 1 receptor (TIR) motif‐containing protein 1 (SARM1) is a pivotal molecule that has garnered extensive attention in neuroscience. As an intracellular molecule, SARM1 possesses various crucial biological functions in the nervous system.
Junjie Wang, Yuhang Shi, Jinglei Tian, Liming Tang, Fang Cao   +4 more
wiley   +1 more source

Hemophilia gene therapy knowledge and perceptions: Results of an international survey

Research and Practice in Thrombosis and Haemostasis, 2020
Background Hemophilia gene therapy is a rapidly evolving therapeutic approach in which a number of programs are approaching clinical development completion.
Flora Peyvandi, David Lillicrap, Johnny Mahlangu, Claire McLintock, K. John Pasi, Steven W. Pipe, Wendy Scales, Alok Srivastava, Thierry VandenDriessche   +8 more
doaj   +1 more source

Evaluation of coagulation disorders and iron deficiency in women with heavy menstrual bleeding

International Journal of Gynecology &Obstetrics, EarlyView.
Abstract Objective To explore the incidence of congenital bleeding disorders (CBD), which may result in anemia, in a large cohort of women referred for heavy menstrual bleeding (HMB) in a specialized gynecologic unit. Methods Between January 2022 and January 2024, all women referred for HMB were screened.
Lucia Rugeri, Yelena Fruchou, Caroline Brodbeck, Marianne Bastien, Amandine Lioux, Giulia Gouy, Marion Cortet, Gil Dubernard, Yesim Dargaud   +8 more
wiley   +1 more source

Acquired Haemophilia Occurring in Association With Bullous Pemphigoid

JEADV Clinical Practice, EarlyView.
ABSTRACT Acquired haemophilia A (AHA) is a rare condition due to neutralizing antibodies against Factor VIII and can result in severe bleeding manifestations. The association of AHA with bullous pemphigoid (BP) is rare, most frequently observed in the elderly, and has been attributed to cross‐reactive antibodies targeting Factor VIII and BPAG2 protein.
Wen Yang Benjamin Ho, Zi Teng Chai, Haur Yueh Lee, Yen Loo Lim   +3 more
wiley   +1 more source