Results 61 to 70 of about 13,284,040 (264)

Nonacog beta pegol (N9‐GP) in hemophilia B: First report on safety and efficacy in previously untreated and minimally treated patients

Research and Practice in Thrombosis and Haemostasis, 2020
We report the first analysis of an extended half‐life recombinant factor IX, nonacog beta pegol (N9‐GP), in previously untreated patients (PUPs) and minimally treated patients with hemophilia B.
Anthony K. C. Chan, J. Alamelu, C. Barnes, A. Chuansumrit, M. Garly, R. M. Meldgaard, G. Young   +6 more
semanticscholar   +1 more source

Recurrent bleeding after rubber band ligation diagnosed as mild hemophilia B: a case report and literature review

BMC Surgery, 2022
Background Hemophilia is a recessive hemorrhagic disease relevant to X chromosome. In mild hemophilia cases, spontaneous bleeding is rare and the blood clotting function is normal, but severe bleeding may occur after trauma or surgery.
Xiaoying Jiang, Min Xu, Yaqing Ding, Yongqing Cao, Yibin Pan   +4 more
doaj   +1 more source

Effects of Social Capital on Career Development Competency Among Young People With Chronic Disabling Health Conditions: Mediating Role of Chronic Illness Self‐Management

Journal of Employment Counseling, EarlyView.
ABSTRACT This study explores how social capital and chronic illness self‐management influence career development competency among young people with chronic disabling health conditions (YPCDHC) in Hong Kong. Using data from 485 participants aged 15–29, path analysis showed that school/workplace social capital had the strongest positive effects on career
Steven Sek‐yum Ngai, Chau‐kiu Cheung, Yuen‐hang Ng, Pamela Pui‐yu Leung, Mike Kwun‐ting Cheung, Hanlei Du, Haoyi Guo, Yongjia He, Hiu‐lam Ngai, Laing‐ming Wong, Qiushi Zhou   +10 more
wiley   +1 more source

Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing "Empty Viral Particles" on Safety and Efficacy of Gene Transfer

Blood, 2018
Background: We have previously shown that a single intravenous administration of a self-complementary adeno-associated virus (scAAV) vector containing a codon-optimised factor IX gene, under control of a synthetic liver specific promoter and ...
A. Nathwani, U. Reiss, E. Tuddenham, P. Chowdary, J. McIntosh, A. Riddell, J. Pie, Johnny Mahlangu, M. Recht, Yu-min P Shen, K. Halka, M. Meagher, A. Nienhuis, A. Davidoff, S. Mangles, C. Morton, Junfang Zhou, V. Radulescu   +17 more
semanticscholar   +1 more source

Patients with obesity have a higher risk of periprosthetic joint infection and more frequent polymicrobial infections after knee arthroplasty: A nationwide register‐based study from Denmark

Knee Surgery, Sports Traumatology, Arthroscopy, EarlyView.
Abstract Purpose To compare the 2‐year incidence of (1) revision due to periprosthetic joint infection (PJI) and (2) all‐cause revision after primary knee arthroplasty (KA) in patients with/without obesity and to compare the microbial profiles in early (≤ 90 days) and late (91–730 days) PJI.
Saber M. Aljuboori, Espen Jimenez‐Solem, Henrik Calum, Robin Christensen, Søren Overgaard   +4 more
wiley   +1 more source

Successful correction of hemophilia by CRISPR/Cas9 genome editing in vivo: delivery vector and immune responses are the key to success

EMBO Molecular Medicine, 2016
Hemophilia B is a serious hemostasis disorder due to mutations of the factor IX gene in the X chromosome. Gene therapy has gained momentum in recent years as a therapeutic option for hemophilia B.
Tuan Huy Nguyen, Ignacio Anegon
doaj   +1 more source

Prime editing in neuropsychiatric disorders: From mutation‐specific target selection to clinical translation

Neuroprotection, EarlyView.
Abstract Prime editing, a novel clustered regularly interspaced short palindromic repeats (CRISPR)‐based technology, fuses a reverse transcriptase (RT) to an engineered CRISPR‐associated protein 9 (Cas9) and uses a prime editing guide RNA (pegRNA)‐encoded template.
Tianshan Ji, Yuan Zhang, Jinyi Zhao, Yiping Lu, Chengkun Wang   +4 more
wiley   +1 more source

Long-term correction of hemophilia B using adenoviral delivery of CRISPR/Cas9

Journal of Controlled Release, 2019
Hemophilia B (HB) is a life‐threatening inherited disease caused by mutations in the FIX gene, leading to reduced protein function and abnormal blood clotting. Due to its monogenic nature, HB is one of the primary targets for gene therapy.
C. Stephens, Elvin J Lauron, E. Kashentseva, Z. Lu, W. Yokoyama, D. Curiel   +5 more
semanticscholar   +1 more source

Red lines and green lights: Gene therapy for inherited erythroid disorders beyond the haemoglobinopathies

British Journal of Haematology, EarlyView.
Gene therapy is revolutionizing treatment paradigms for haemoglobinopathies, establishing a translational framework for disorders that impact red blood cell development. In their paper, Joshi et al. describe the preclinical and early clinical landscape of gene therapies for non‐haemoglobinopathy erythroid disorders and highlight common thematic ...
Gaurav Joshi, Nandakumar Natarajan, Kathryn E. Dickerson, Richard A. Voit   +3 more
wiley   +1 more source

Strategies for Hemophilia Treatment, a literature review of current evidence

مجله دانشگاه علوم پزشکی بیرجند, 2023
Hemophilia is an inherited bleeding disorder caused by malfunctioning or lacking blood coagulation factor VIII (hemophilia A) or IX (hemophilia B). Currently, the main treatments for these X-linked diseases are replacement therapy using periodic and ...
Fahimeh Ghasemi, Nafiseh Erfanian, Samira Karbasi, Alireza Zomorodipour   +3 more
doaj