Results 11 to 20 of about 69,645 (303)

Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B.

New England Journal of Medicine, 2023
BACKGROUND Moderate-to-severe hemophilia B is treated with lifelong, continuous coagulation factor IX replacement to prevent bleeding. Gene therapy for hemophilia B aims to establish sustained factor IX activity, thereby protecting against bleeding ...
S. Pipe, F. Leebeek, M. Recht, N. Key, G. Castaman, Wolfgang A. Miesbach, S. Lattimore, K. Peerlinck, P. R. van der Valk, M. Coppens, P. Kampmann, K. Meijer, Niamh O'Connell, K. Pasi, D. Hart, R. Kazmi, J. Astermark, C. Hermans, R. Klamroth, Richard W. Lemons, N. Visweshwar, Annette von Drygalski, G. Young, S. Crary, M. Escobar, E. Gomez, R. Kruse-Jarres, D. Quon, E. Symington, Michael Wang, Allison P. Wheeler, R. Gut, Yinyin Liu, R. Dolmetsch, D. Cooper, Yanyan Li, B. Goldstein, P. Monahan   +37 more
semanticscholar   +1 more source

Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A.

New England Journal of Medicine, 2023
BACKGROUND Valoctocogene roxaparvovec delivers a B-domain-deleted factor VIII coding sequence with an adeno-associated virus vector to prevent bleeding in persons with severe hemophilia A.
J. Mahlangu, R. Kaczmarek, Annette von Drygalski, S. Shapiro, Sheng-Chieh Chou, M. Ozelo, G. Kenet, F. Peyvandi, Michael Wang, B. Madan, N. Key, Michael Laffan, A. Dunn, J. Mason, D. Quon, E. Symington, A. Leavitt, J. Oldenburg, H. Chambost, M. Reding, K. Jayaram, Hua Yu, Reena Mahajan, K. Chavele, D. B. Reddy, Joshua Henshaw, T. Robinson, W. Y. Wong, S. Pipe   +28 more
semanticscholar   +1 more source

Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A.

New England Journal of Medicine, 2022
BACKGROUND Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is an adeno-associated virus 5 (AAV5)-based gene-therapy vector containing a coagulation factor VIII complementary DNA driven by a liver-selective promoter.
M. Ozelo, J. Mahlangu, K. Pasi, A. Giermasz, A. Leavitt, M. Laffan, E. Symington, D. Quon, Jiaan-Der Wang, K. Peerlinck, S. Pipe, B. Madan, N. Key, G. Pierce, Brian O'Mahony, R. Kaczmarek, Joshua Henshaw, A. Lawal, K. Jayaram, Mei Huang, Xinqun Yang, W. Y. Wong, Benjamin Kim   +22 more
semanticscholar   +1 more source

In vivo delivery of CRISPR-Cas9 using lipid nanoparticles enables antithrombin gene editing for sustainable hemophilia A and B therapy

Science Advances, 2022
Hemophilia is a hereditary disease that remains incurable. Although innovative treatments such as gene therapy or bispecific antibody therapy have been introduced, substantial unmet needs still exist with respect to achieving long-lasting therapeutic ...
J. Han, MinJeong Kim, B. Choi, Jeong Hyeon Lee, G. Lee, M. Jeong, Yeji Lee, Eun-Ah Kim, Hye-Kyung Oh, Nanyeong Go, Hyerim Lee, K. J. Lee, U. Kim, J. Y. Lee, Seokjoong Kim, Jun Chang, Hyukjin Lee, D. Song, S. Yeom   +18 more
semanticscholar   +1 more source

The Hemophilia Joint Health Score version 2.1 Validation in Adult Patients Study: A multicenter international study

Research and Practice in Thrombosis and Haemostasis, 2022
The Hemophilia Joint Health Score (HJHS) was developed and validated to detect arthropathy in children. Additional evidence is required to show validity in adults.
J. St-Louis, A. Abad, S. Funk, M. Tilak, S. Classey, N. Zourikian, P. Mclaughlin, S. Lobet, Grace Hernandez, S. Akins, A. Wells, M. Manco-Johnson, J. John, S. Austin, Pratima Chowdhary, C. Hermans, D. Nugent, N. Bakeer, S. Mangles, P. Hilliard, V. Blanchette, B. Feldman   +21 more
semanticscholar   +1 more source

Adeno-associated Virus Gene Therapy for Hemophilia.

Annual Review of Medicine, 2022
In vivo gene therapy is rapidly emerging as a new therapeutic paradigm for monogenic disorders. For almost three decades, hemophilia A (HA) and hemophilia B (HB) have served as model disorders for the development of gene therapy.
B. Samelson-Jones, Lindsey A. George
semanticscholar   +1 more source

WFH Guidelines for the Management of Hemophilia, 3rd edition

Haemophilia, 2020
Alok Srivastava 1 | Elena Santagostino 2 | Alison Dougall 3 | Steve Kitchen 4 | Megan Sutherland 5 | Steven W. Pipe 6 | Manuel Carcao 7 | Johnny Mahlangu 8 | Margaret V. Ragni 9 | Jerzy Windyga 10 | Adolfo Llinás 11 | Nicholas J. Goddard 12 | Richa Mohan
A. Srivastava, E. Santagostino, A. Dougall, S. Kitchen, M. Sutherland, S. Pipe, M. Carcao, J. Mahlangu, M. Ragni, J. Windyga, A. Llinás, N. Goddard, Richa Mohan, P. Poonnoose, B. Feldman, Sandra Zelman Lewis, H. Berg, Glenn F. Pierce   +17 more
semanticscholar   +1 more source

Gene therapy for hemophilia.

Hematology. American Society of Hematology. Education Program, 2022
The cloning of the factor VIII (FVIII) and factor IX (FIX) genes in the 1980s has led to a succession of clinical advances starting with the advent of molecular diagnostic for hemophilia, followed by the development of recombinant clotting factor ...
A. Nathwani
semanticscholar   +1 more source

Prophylactic emicizumab for hemophilia A in the Asia‐Pacific region: A randomized study (HAVEN 5)

Research and Practice in Thrombosis and Haemostasis, 2022
Emicizumab is a subcutaneously administered humanized, bispecific, monoclonal antibody approved for prophylaxis in people with hemophilia A.
Renchi Yang, Shujie Wang, Xuefeng Wang, Jing Sun, A. Chuansumrit, Jianfeng Zhou, C. Schmitt, W. Hsu, Jeffrey Xu, Lindong Li, Tiffany Y. Chang, Xielan Zhao   +11 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