Results 21 to 30 of about 22,597,991 (289)

Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A.

New England Journal of Medicine, 2020
BACKGROUND Adeno-associated virus (AAV)-mediated gene therapy is under investigation as a therapeutic option for persons with hemophilia A. Efficacy and safety data include 3 years of follow-up after a single administration of AAV5-hFVIII-SQ.
K. Pasi, S. Rangarajan, N. Mitchell, W. Lester, E. Symington, B. Madan, M. Laffan, C. Russell, Mingjin Li, Glenn F Pierce, W. Y. Wong   +10 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

A factor VIIIa–mimetic bispecific antibody, Mim8, ameliorates bleeding upon severe vascular challenge in hemophilia A mice

Blood, 2021
Østergaard and colleagues report the development of a potent bispecific antibody, Mim8, which binds both activated factor IX and factor X, mimicking the propagation of coagulation by activated factor VIII.
Henrik Østergaard, J. Lund, Per Greisen, S. Kjellev, A. Henriksen, Nikolai Lorenzen, E. Johansson, G. Røder, M. G. Rasch, L. Johnsen, T. Egebjerg, S. Lund, H. Rahbek-Nielsen, Prafull S Gandhi, K. Lamberth, M. Loftager, L. Andersen, A. C. Bonde, F. Stavenuiter, D. E. Madsen, Xun Li, T. L. Holm, C. D. Ley, P. Thygesen, Haisun Zhu, Rong Zhou, Karina Thorn, Zhiru Yang, M. B. Hermit, J. Bjelke, B. G. Hansen, I. Hilden   +31 more
semanticscholar   +1 more source

International recommendations on the diagnosis and treatment of acquired hemophilia A

Haematologica, 2020
Acquired hemophilia A (AHA), a rare bleeding disorder caused by neutralizing autoantibodies against coagulation factor VIII (FVIII), occurs in both men and women without a previous history of bleeding.
A. Tiede, P. Collins, P. Knoebl, J. Teitel, C. Kessler, M. Shima, G. Di Minno, R. d’Oiron, P. Salaj, V. Jiménez‐Yuste, A. Huth‐Kühne, P. Giangrande   +11 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

Recent Advances in the Treatment of Hemophilia: A Review

Biologics : targets & therapy, 2021
Progress in hemophilia therapy has been remarkable in the first 20 years of the third millennium, but the innovation began with the description the fractionation of plasma in 1946.
E. Marchesini, M. Morfini, L. Valentino
semanticscholar   +1 more source

In Utero Transplantation of Placenta-Derived Mesenchymal Stromal Cells for Potential Fetal Treatment of Hemophilia A. [PDF]

, 2018
Hemophilia A (HA) is an X-linked recessive disorder caused by mutations in the factor VIII ( FVIII) gene leading to deficient blood coagulation. The current standard of care is frequent infusions of plasma-derived FVIII or recombinant B-domain-deleted ...
Farmer, Diana, Gao, Kewa, Kumar, Priyadarsini, Lankford, Lee, Pivetti, Christopher, Wang, Aijun, Wang, Chuwang   +6 more
core   +1 more source

A long-term study of AAV gene therapy in hemophilia A dogs identifies clonal expansions of transduced liver cells

Nature Biotechnology, 2020
Nine dogs with hemophilia A were treated with adeno-associated viral (AAV) gene therapy and followed for up to 10 years. Administration of AAV8 or AAV9 vectors expressing canine factor VIII (AAV-cFVIII) corrected the FVIII deficiency to 1.9–11.3% of ...
Giang N. Nguyen, J. Everett, Samita Kafle, A. Roche, H. Raymond, Jacob S. Leiby, Christian Wood, C. Assenmacher, E. Merricks, C. T. Long, H. Kazazian, T. Nichols, F. Bushman, D. Sabatino, D. Sabatino   +14 more
semanticscholar   +1 more source

Haemophilia and joint disease: pathophysiology, evaluation and management [PDF]

, 2011
In patients with haemophilia, regular replacement therapy with clotting factor concentrates (prophylaxis) is effective in preventing recurrent bleeding episodes into joints and muscles.
Berntorp, Erik, Knobe, Karin
core   +3 more sources

Incidence and mortality rates of intracranial hemorrhage in hemophilia: a systematic review and meta-analysis.

Blood, 2021
Intracranial hemorrhage (ICH) is a severe complication that is relatively common among hemophilia patients. This systematic review aimed to obtain more precise estimates of ICH incidence and mortality in hemophilia, which may be important for patients ...
A. Zwagemaker, S. Gouw, Julie J Jansen, C. Vuong, M. Coppens, Q. Hu, Xiao-qin Feng, S. Kim, J. G. van der Bom, K. Fijnvandraat   +9 more
semanticscholar   +1 more source