Results 101 to 110 of about 12,981,488 (234)

Hemophilia B Gene Therapy with a High‐Specific‐Activity Factor IX Variant

New England Journal of Medicine, 2017
Lindsey A. George, S. Sullivan, A. Giermasz, J. Rasko, B. Samelson-Jones, J. Ducore, A. Cuker, L. Sullivan, S. Majumdar, J. Teitel, C. McGuinn, M. Ragni, A. Luk, Daniel J. Hui, Fraser Wright, Yifeng Chen, Yun Liu, Katie Wachtel, Angela Winters, S. Tiefenbacher, V. Arruda, J. C. M. van der Loo, O. Zelenaia, D. Takefman, M. Carr, L. Couto, X. Anguela, K. High   +27 more
semanticscholar   +1 more source

Molecular diagnosis of hemophilia A and B. Report of five families from Costa Rica

Revista de Biología Tropical, 2004
Hemophilia Aand B are X-chromosome linked bleeding disorders caused by deficiency of the respective coagulation factor VIII and IX. Affected individuals develop a variable phenotype of hemorrhage caused by a broad range of mutations within the Factor ...
Lizbeth Salazar-Sánchez, Guillermo Jiménez-Cruz, Pilar Chaverri, Winnie Schröder, Karin Wulff, Gerardo Jiménez-Arce, Miriam Sandoval, Patricia Ramírez, F.H Herrmann   +8 more
doaj  

Evaluating the Safety and Efficacy of Concizumab in Hemophilia A/B Patients: A Systematic Review

Clinical and Applied Thrombosis/Hemostasis
Background Hemophilia A and B are X-linked recessive bleeding disorders caused by deficiencies of coagulation factors VIII and IX, respectively. These conditions lead to spontaneous bleeding, joint damage, inhibitor development, and the burden of ...
Erum Siddiqui MBBS, Maliha Khalid MBBS, Muhammad Saad Khan MBBS, Kanza Farhan MBBS, Muhammad Mohsin Khan MBBS, Aminath Waafira MBBS   +5 more
doaj   +1 more source

Discussion:Future Considerations in the Management of Hemophilia B [PDF]

, 1995

openalex   +1 more source

Nine independentF9 mutations in the Mexican hemophilia B population: Nonrandom recurrences of point mutation events in the human germline [PDF]

, 2000
Ana Rebeca Jaloma‐Cruz, William A. Scaringe, Joni B. Drost, Stacy Roberts, Xuemin Li, Patricio Barros-N��ez, Luis E. Figuera, Fernando Rivas, Jos� Mar�a Cant�, Steve S. Sommer   +9 more
openalex   +1 more source

Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B.

Molecular Therapy, 2020
Lindsey A. George, M. Ragni, J. Rasko, L. Raffini, B. Samelson-Jones, M. Ozelo, Maria Hazbon, Alexa R. Runowski, J. Wellman, Katie Wachtel, Yifeng Chen, X. Anguela, K. Kuranda, F. Mingozzi, K. High   +14 more
semanticscholar   +1 more source

Molecular basis of hemophilia B: a defective enzyme due to an unprocessed propeptide is caused by a point mutation in the factor IX precursor.

, 1986
David Diuguid, Marie‐Josèphe Rabiet, Bruce Furie, Howard A. Liebman, Bruce Furie   +4 more
openalex   +1 more source

Muscle-Directed Gene Transfer and Transient Immune Suppression Result in Sustained Partial Correction of Canine Hemophilia B Caused by a Null Mutation [PDF]

, 2001
Roland W. Herzog, Jane Mount, Valder R. Arruda, Katherine A. High, Clinton D. Lothrop   +4 more
openalex   +1 more source

Sustained and Complete Phenotype Correction of Hemophilia B Mice Following Intramuscular Injection of AAV1 Serotype Vectors [PDF]

, 2001
Hengjun Chao, Paul E. Monahan, Yuanbo Liu, R. Jude Samulski, Christopher Walsh   +4 more
openalex   +1 more source

250. High Level Expression of Canine Factor IX in Hemophilia B Dogs after Readminstration of AAV2/5 Vectors [PDF]

, 2002

openalex   +1 more source