Results 41 to 50 of about 13,284,040 (264)

Neonatal Gene Therapy for Hemophilia B by a Novel Adenovirus Vector Showing Reduced Leaky Expression of Viral Genes

Molecular Therapy: Methods & Clinical Development, 2017
Gene therapy during neonatal and infant stages is a promising approach for hemophilia B, a congenital disorder caused by deficiency of blood coagulation factor IX (FIX).
Shunsuke Iizuka, Fuminori Sakurai, Masashi Tachibana, Kazuo Ohashi, Hiroyuki Mizuguchi   +4 more
doaj   +1 more source

Etranacogene dezaparvovec for hemophilia B gene therapy

Therapeutic Advances in Rare Disease, 2021
The treatment landscape for hemophilia has been rapidly changing with introduction of novel therapies. Gene therapy for hemophilia is a promising therapeutic option for sustained endogenous factor production to mitigate the need for prophylactic ...
C. Thornburg
semanticscholar   +1 more source

Haemophilia B: an illustrative review of current challenges and opportunities

Research and Practice in Thrombosis and Haemostasis
Background: Hemophilia B is a genetic bleeding disorder caused by a deficiency of clotting factor IX, which presents unique challenges in clinical management.
Cedric Hermans, Jan Astermark, Sonata Šaulytė Trakymienė, Víctor Jiménez-Yuste   +3 more
doaj   +1 more source

Hemophilia B and gene therapy: a new chapter with etranacogene dezaparvovec

Blood Advances
The US Food and Drug Administration (FDA)’s authorization of etranacogene dezaparvovec (Hemgenix) is a significant milestone, constituting not only the first FDA approval of a gene therapy for hemophilia but also the first approval of a liver-targeted ...
X. Anguela, Katherine A. High
semanticscholar   +1 more source

Therapeutic hFIX Activity Achieved after Single AAV5-hFIX Treatment in Hemophilia B Patients and NHPs with Pre-existing Anti-AAV5 NABs

Molecular Therapy: Methods & Clinical Development, 2019
Currently, individuals with pre-existing neutralizing antibodies (NABs) against adeno-associated virus (AAV) above titer of 5 are excluded from systemic AAV-based clinical trials. In this study we explored the impact of pre-existing anti-AAV5 NABs on the
A. Majowicz, B. Nijmeijer, M. Lampen, Lisa Spronck, M. de Haan, H. Petry, S. V. van Deventer, C. Meyer, M. Tangelder, Valérie Ferreira   +9 more
semanticscholar   +1 more source

Protein-Engineered Coagulation Factors for Hemophilia Gene Therapy

Molecular Therapy: Methods & Clinical Development, 2019
Hemophilia A (HA) and hemophilia B (HB) are X-linked bleeding disorders due to inheritable deficiencies in either coagulation factor VIII (FVIII) or factor IX (FIX), respectively.
Benjamin J. Samelson-Jones, Valder R. Arruda   +1 more
doaj   +1 more source

Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B.

Blood Advances, 2019
Etranacogene dezaparvovec (AMT-061) is a recombinant AAV5 vector including a gene cassette containing the factor IX (FIX) Padua variant under the control of a liver-specific promoter.
Annette von Drygalski, A. Giermasz, G. Castaman, N. Key, S. Lattimore, F. Leebeek, Wolfgang A. Miesbach, M. Recht, A. Long, R. Gut, E. Sawyer, S. Pipe   +11 more
semanticscholar   +1 more source

CRISPR-Cas9-Mediated In Vivo Gene Integration at the Albumin Locus Recovers Hemostasis in Neonatal and Adult Hemophilia B Mice

Molecular Therapy: Methods & Clinical Development, 2020
Clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 loaded by vectors could induce high rates of specific site genome editing and correct disease-causing mutations.
Qingnan Wang, Xiaomei Zhong, Qian Li, Jing Su, Yi Liu, Li Mo, H. Deng, Yang Yang   +7 more
semanticscholar   +1 more source

T2* Magnetic Resonance Imaging Uncovers Hemosiderin Burden in Pediatric Hemophilia: A Call for Sensitive Imaging Biomarkers

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Hemophilic arthropathy remains the leading morbidity in hemophilia despite modern prophylaxis, and early joint damage may be missed by routine exams. This study explored T2* MRI as a noninvasive biomarker of hemosiderin deposition in pediatric hemophilia.
Jessica Garcia, Tarique Hussain, Jeanne Dillenbeck, Matthew Hammer, Annette von Drygalski, Guy Young, Ayesha Zia   +6 more
wiley   +1 more source

Long‐term safety and efficacy of rIX‐FP prophylaxis with extended dosing intervals up to 21 days in adults/adolescents with hemophilia B

Journal of Thrombosis and Haemostasis, 2020
An international, multicenter extension study evaluated recombinant fusion protein linking recombinant coagulation factor IX (FIX) with recombinant human albumin (rIX‐FP) in hemophilia B (FIX ≤ 2%) patients previously enrolled in a phase III study or who
M. Mancuso, A. Lubetsky, Brigitte Pan-Petesch, T. Lissitchkov, A. Nagao, W. Seifert, Yanyan Li, E. Santagostino   +7 more
semanticscholar   +1 more source