Results 11 to 20 of about 13,284,040 (264)

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

In vivo genome editing for hemophilia B therapy by the combination of rebalancing and therapeutic gene knockin using a viral and non-viral vector.

Molecular Therapy: Nucleic Acids, 2023
Recent therapeutic strategies for hemophilia include long-term therapeutic gene expression using adeno-associated virus (AAV) and rebalancing therapy via the downregulation of anticoagulant pathways.
J. Lee, J. Han, D. Song, G. Lee, B. Choi, MinJeong Kim, Yeji Lee, Seokjoong Kim, Hyukjin Lee, S. Yeom   +9 more
semanticscholar   +1 more source

Phase 1-2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B.

New England Journal of Medicine, 2022
BACKGROUND FLT180a (verbrinacogene setparvovec) is a liver-directed adeno-associated virus (AAV) gene therapy that uses a synthetic capsid and a gain-of-function protein to normalize factor IX levels in patients with hemophilia B.
P. Chowdary, S. Shapiro, M. Makris, G. Evans, S. Boyce, K. Talks, G. Dolan, U. Reiss, M. Phillips, A. Riddell, M. R. Peralta, M. Quaye, D. W. Patch, E. Tuddenham, A. Dane, M. Watissée, A. Long, A. Nathwani   +17 more
semanticscholar   +1 more source

Managing Relevant Clinical Conditions of Hemophilia A/B Patients

Hematology Reports, 2023
The Medical Directors of nine Italian Hemophilia Centers reviewed and discussed the key issues concerning the replacement therapy of hemophilia patients during a one-day consensus conference held in Rome one year ago. Particular attention was paid to the
Massimo Morfini, Jacopo Agnelli Giacchiello, Erminia Baldacci, Christian Carulli, Giancarlo Castaman, Anna Chiara Giuffrida, Giuseppe Malcangi, Angiola Rocino, Sergio Siragusa, Ezio Zanon   +9 more
doaj   +1 more source

PAM-flexible Cas9-mediated base editing of a hemophilia B mutation in induced pluripotent stem cells

Communications Medicine, 2023
Background Base editing via CRISPR-Cas9 has garnered attention as a method for correcting disease-specific mutations without causing double-strand breaks, thereby avoiding large deletions and translocations in the host chromosome.
Takafumi Hiramoto, Y. Kashiwakura, M. Hayakawa, N. Baatartsogt, N. Kamoshita, Tomoyuki Abe, H. Inaba, H. Nishimasu, Hideki Uosaki, Y. Hanazono, O. Nureki, T. Ohmori   +11 more
semanticscholar   +1 more source

Gene Therapy Approaches for the Treatment of Hemophilia B

International Journal of Molecular Sciences, 2023
In contrast to the standard enzyme-replacement therapy, administered from once per 7–14 days to 2–3 times a week in patients with severe hemophilia B, as a result of a single injection, gene therapy can restore F9 gene expression and maintain it for a ...
Anastasiia B. Soroka, S. Feoktistova, O. Mityaeva, P. Volchkov   +3 more
semanticscholar   +1 more source

Adeno‐associated virus serotype 2 capsid variants for improved liver‐directed gene therapy

Hepatology, EarlyView., 2022
Abstract Background and Aims Current liver‐directed gene therapies look for adeno‐associated virus (AAV) vectors with improved efficacy. With this background, capsid engineering is explored. Whereas shuffled capsid library screenings have resulted in potent liver targeting variants with one first vector in human clinical trials, modifying natural ...
Nadja Meumann, Marti Cabanes‐Creus, Moritz Ertelt, Renina Gale Navarro, Julie Lucifora, Qinggong Yuan, Karin Nien‐Huber, Ahmed Abdelrahman, Xuan‐Khang Vu, Liang Zhang, Ann‐Christin Franke, Christian Schmithals, Albrecht Piiper, Annabelle Vogt, Maria Gonzalez‐Carmona, Jochen T. Frueh, Evelyn Ullrich, Philip Meuleman, Steven R. Talbot, Margarete Odenthal, Michael Ott, Erhard Seifried, Clara T. Schoeder, Joachim Schwäble, Leszek Lisowski, Hildegard Büning   +25 more
wiley   +1 more source

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

First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia B

Molecular Therapy, 2022
Zinc-finger nuclease (ZFN)-based in vivo genome editing is a novel treatment that can potentially provide lifelong protein replacement with single intravenous administration.
P. Harmatz, C. Prada, B. Burton, H. Lau, C. Kessler, L. Cao, M. Falaleeva, A. G. Villegas, Jennifer Zeitler, Kathleen Meyer, Weston P. Miller, C. W. P. Foo, S. Vaidya, W. Swenson, Lisa H. Shiue, D. Rouy, J. Muenzer   +16 more
semanticscholar   +1 more source

Comprehensive analysis and prediction of long-term durability of factor IX activity following etranacogene dezaparvovec gene therapy in the treatment of hemophilia B

Current Medical Research and Opinion, 2022
Objective Congenital hemophilia B is a rare bleeding disorder caused by defects in the gene encoding factor IX (FIX) leading to coagulation deficiency. Recurrent bleeds may cause chronic pain, disability, and reduced quality of life.
Jinesh Shah, Hongseok Kim, K. Sivamurthy, P. Monahan, Michael Fries   +4 more
semanticscholar   +1 more source