Results 21 to 30 of about 13,086,034 (342)

Seroprevalence to adeno‐associated virus type 6 in people with hemophilia B from a UK adult cohort

Research and Practice in Thrombosis and Haemostasis, 2022
Background Gene therapy shows promise as a potential “cure” for hemophilia A and B. Adeno‐associated virus (AAV) vectors are the leading platform to deliver modified genetic code of factor VIII or IX to the liver effecting endogenous production.
Sara Boyce, Izabela James, Savita Rangarajan, Nicola Curry, Catherine Bagot, Steven Austin, Mike Laffan, Sarah Mangles, Kandiah Chandrakumaran, Carina Mundy   +9 more
doaj   +1 more source

Insights into the Molecular Genetic of Hemophilia A and Hemophilia B: The Relevance of Genetic Testing in Routine Clinical Practice

Hämostaseologie, 2022
Hemophilia A and hemophilia B are rare congenital, recessive X-linked disorders caused by lack or deficiency of clotting factor VIII (FVIII) or IX (FIX), respectively.
B. Pezeshkpoor, J. Oldenburg, A. Pavlova
semanticscholar   +1 more source

A Case of Acquired Hemophilia A and Congenital Hemophilia B

Cureus, 2022
Congenital hemophilia B is a rare, inherited X-linked bleeding disorder caused by a deficiency of factor IX (FIX). Acquired hemophilia A is a rare, acquired bleeding disorder which presents as new onset bleeding in older adults due to the development of autoantibodies against factor VIII (FVIII).
Fortier, Julia C, Pang, Shiyi S, Amofa-Ho, Sam, Harris, Neil S, Zumberg, Marc   +4 more
openaire   +2 more sources

Low-dose AAV-CRISPR-mediated liver-specific knock-in restored hemostasis in neonatal hemophilia B mice with subtle antibody response

Nature Communications, 2022
AAV-delivered CRISPR/Cas9 (AAV-CRISPR) has shown promising potentials in preclinical models to efficiently insert therapeutic gene sequences in somatic tissues.
Xiangjun He, Zhenjie Zhang, Jun Xue, Yaofeng Wang, Siqi Zhang, Junkang Wei, Chenzi Zhang, Jue Wang, Brian Anugerah Urip, Chun Christopher Ngan, Junjiang Sun, Yuefeng Li, Zhiqian Lu, Hui Zhao, D. Pei, Chi-kong Li, Bo Feng   +16 more
semanticscholar   +1 more source

Final results of the PUPs B-LONG study: evaluating safety and efficacy of rFIXFc in previously untreated patients with hemophilia B.

Blood Advances, 2021
PUPs B-LONG evaluated the safety and efficacy of recombinant factor IX Fc fusion protein (rFIXFc) in previously untreated patients (PUPs) with hemophilia B. In this open-label, phase 3 study, male PUPs (age
B. Nolan, A. Klukowska, A. Shapiro, A. Rauch, M. Recht, M. Ragni, J. Curtin, S. Gunawardena, Sutirtha Mukhopadhyay, D. Jayawardene, B. Winding, K. Fischer, R. Liesner   +12 more
semanticscholar   +1 more source

The Cost-effectiveness of Gene Therapy for Severe Hemophilia B: Microsimulation Study from the United States Perspective.

Blood, 2021
Adeno-associated virus (AAV)-mediated gene therapy is a novel treatment promising to reduce morbidity associated with hemophilia. While multiple clinical trials continue to evaluate efficacy and safety, limited cost-effectiveness data have been published.
N. Bolous, Yichen Chen, Huiqi Wang, A. Davidoff, M. Devidas, Timothy W. Jacobs, M. Meagher, A. Nathwani, E. Neufeld, Bryan A. Piras, C. Rodríguez-Galindo, U. Reiss, N. Bhakta   +12 more
semanticscholar   +1 more source

Health care resource utilization and cost burden of hemophilia B in the United States.

Blood Advances, 2021
Hemophilia B is a rare congenital blood disorder characterized by factor IX deficiency. Clinical profiles of hemophilia B range from mild to severe forms of the disease.
T. Buckner, I. Bocharova, K. Hagan, A. Bensimon, Hongbo Yang, E. Wu, E. Sawyer, Nanxin Li   +7 more
semanticscholar   +1 more source

Inhibitors in Patients with Congenital Bleeding Disorders Other Than Hemophilia [PDF]

, 2017
The most worrying complication of replacement therapy for severe hemophilia A and B is currently the occurrence of inhibitory alloantibodies against infused factor VIII and factor IX, respectively.
Franchini, Massimo, Liumbruno, Giancarlo Maria, Marano, Giuseppe, Mengoli, Carlo, Piccinini, Vanessa, Pupella, Simonetta, Vaglio, Stefania   +6 more
core   +1 more source

Identification and Genetic Analysis of a Factor IX Gene Intron 3 Mutation in a Hemophilia B Pedigree in China

Turkish Journal of Hematology, 2014
OBJECTIVE: Hemophilia B is caused by coagulation defects in the factor IX gene located in Xq27.1 on the X chromosome. A wide range of mutations, showing extensive molecular heterogeneity, have been described in hemophilia B patients.
Dong Hua Cao, Xiao Li Liu, Kai Mu, Xiang Wei Ma, Jing Li Sun, Xiao Zhong Bai, Chang Kun Lin, Chun Lian Jin   +7 more
doaj   +1 more source

BAX 335 hemophilia B gene therapy clinical trial results - potential impact of CpG sequences on gene expression.

Blood, 2020
Gene therapy has the potential to maintain therapeutic blood clotting factor IX (FIX) levels in patients with hemophilia B by delivering a functional human F9 gene into liver cells.
B. Konkle, C. Walsh, Miguel A. Escobar, Neil C Josephson, G. Young, Annette von Drygalski, S. McPhee, R. Samulski, I. Bilic, Maurus de la Rosa, B. Reipert, H. Rottensteiner, F. Scheiflinger, J. Chapin, Bruce M. Ewenstein, P. Monahan   +15 more
semanticscholar   +1 more source