Results 31 to 40 of about 22,643,381 (355)

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

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

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

Medical costs and hospital utilization for hemophilia A and B urban inpatients in China: a national cross-sectional study

BMC Health Services Research, 2022
Background Hemophilia care in mainland China has been greatly improved since the establishment of the Hemophilia Treatment Center Collaborative Network of China (HTCCNC), and most of drugs for hemophilia have been covered by basic medical insurance ...
Zhengwei Huang, Stephen Nicholas, Yong Yang, Xiaoping Chen, Elizabeth Maitland, Yong Ma, Xuefeng Shi   +6 more
doaj   +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

A Rare Case of Acquired Hemophilia A in Adolescents and Young Adults

Clinical Pediatric Hematology-Oncology, 2022
Acquired hemophilia A (AHA) is a rare bleeding disorder, especially in adolescents and young adults (AYAs) attributable to the development of autoantibodies against coagulation factor VIII (FVIII). AHA diagnosis is difficult; patients lack any history of
Min Jeong Lee, Young Shil Park
doaj   +1 more source

Turoctocog alfa pegol (N8‐GP) in severe hemophilia A: Long‐term safety and efficacy in previously treated patients of all ages in the pathfinder8 study

Research and Practice in Thrombosis and Haemostasis, 2022
Background N8‐GP (turoctocog alfa pegol; Esperoct) is a glycoPEGylated human recombinant factor VIII (FVIII). Objectives Pathfinder8 (NCT01480180) was a phase 3, multinational, open‐label, nonrandomized trial to investigate the long‐term safety and ...
Steven R. Lentz, Kaan Kavakli, Robert Klamroth, Mudi Misgav, Azusa Nagao, Alberto Tosetto, Pernille Juul Jørgensen, Marek Zak, Laszlo Nemes   +8 more
doaj   +1 more source

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

Practices and challenges for hemophilia management under resource constraints in Thailand

Orphanet Journal of Rare Diseases, 2023
Hemophilia is an inherited bleeding disorder caused by deficiency of a specific coagulation factor. Factor VIII deficiency is responsible for hemophilia A while factor IX deficiency is responsible for hemophilia B. As per the 2020 annual global survey by
Chatphatai Moonla, Darintr Sosothikul, Bunchoo Pongtanakul, Bundarika Suwanawiboon, Chanchai Traivaree, Rungrote Natesirinilkul, Nongnuch Sirachainan, Pantep Angchaisuksiri   +7 more
doaj   +1 more source

Establishment and Evolution of China National Hemophilia Registry

罕见病研究, 2022
Hemophilia is an inherited bleeding disorder and a type of rare disease that is hereditary, lifelong and disabling. The establishment of a National Hemophilia Registry is foundational to treating hemophilia.
XUE Feng, YANG Renchi
doaj   +1 more source