Results 51 to 60 of about 68,905 (210)

Progress in the use of adeno-associated viral vectors for gene therapy [PDF]

, 2004
The development of safe and efficient gene transfer vectors is crucial for the success of gene therapy trials. A viral vector system promising to meet these requirements is based on the apathogenic adeno-associated virus (AAV-2), a member of the ...
Braun-Falco, M., Buning, H., Hallek, M.
core   +1 more source

Hemophilia gene therapy knowledge and perceptions: Results of an international survey

Research and Practice in Thrombosis and Haemostasis, 2020
Background Hemophilia gene therapy is a rapidly evolving therapeutic approach in which a number of programs are approaching clinical development completion.
Flora Peyvandi, David Lillicrap, Johnny Mahlangu, Claire McLintock, K. John Pasi, Steven W. Pipe, Wendy Scales, Alok Srivastava, Thierry VandenDriessche   +8 more
doaj   +1 more source

Hemophilia of orbit

Oman Journal of Ophthalmology, 2009
Hemophilic pseudotumor is an uncommon complication of factor VIII and IX deficiencies in the coagulation cascade and occurs in a wide spectrum of bones and soft tissues. We present a six-year-old boy with hemophilic pseudotumor localized in the right orbit.
Gunaseelan Karunanithi, Pooja Sethi, Sathyanarayana K Reddy, Vivekanandam   +3 more
openaire   +3 more sources

Hemophilia treatment and transfusion medicine

Zhongguo shuxue zazhi
The improvement of hemophilia treatment is attributed to the development of transfusion medicine, and the discovery of cryoprecipitate has opened up a new era of hemophilia treatment, laying the foundation for the development of modern hemophilia ...
FANG Yunhai, CHENG Yan, ZHANG Xinsheng
doaj   +1 more source

Von Willebrand Disease [PDF]

, 2017
Von Willebrand Disease (VWD) is a blood clotting disorder characterized by low levels of the Von Willebrand Factor (VWF) in the blood. VWF is functions to bind platelets and promote clotting of the blood when vascular injury occurs.
Misla David, Ramon
core   +2 more sources

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

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

Clinical perspectives of emerging pathogens in bleeding disorders. [PDF]

, 2006
As a result of immunological and nucleic-acid screening of plasma donations for transfusion-transmissible viruses, and the incorporation of viral reduction processes during plasma fractionation, coagulation-factor concentrates (CFC) are now judged safe ...
Bozzette, Samuel, Diamond, Michael, Koerper, Marion A, Kulkarni, Roshni, Ludlam, Christopher A, Powderly, William G, Ritchie, Bruce, Siegel, Jamie, Simmonds, Peter, Stanley, Samuel, Tapper, Michael L, von Depka, Mario   +11 more
core   +1 more source

On demand treatment and home therapy of hereditary angioedema in Germany - the Frankfurt experience [PDF]

, 2010
Background: Manifestation of acute edema in hereditary angioedema (HAE) is characterized by interindividual and intraindividual variability in symptom expression over time. Flexible therapy options are needed.
Aygören-Pürsün, Emel, Martinez Saguer, Inmaculada, Rusicke, Eva, Klingebiel, Thomas, Kreuz, Wolfhart   +4 more
core   +2 more sources

Comparative Investigation of Thromboelastometry and Thrombin Generation for Patients Receiving Direct Oral Anticoagulants or Vitamin K Antagonists

Diagnostics
Background. Alterations induced by direct oral anticoagulants (DOACs) or vitamin K antagonists (VKAs) to thromboelastometry and thrombin generation are not well defined.
Armando Tripodi, Marco Capecchi, Erica Scalambrino, Marigrazia Clerici, Barbara Scimeca, Pasquale Agosti, Paolo Bucciarelli, Andrea Artoni, Flora Peyvandi   +8 more
doaj   +1 more source