Results 11 to 20 of about 78,600 (311)

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

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

Immune System-Related Genetic Risk Factors for Inhibitory Antibody Development in Patients With Hemophilia: Reviewing an Old Problem From a New Perspective-A Narrative Review. [PDF]

Health Sci Rep
ABSTRACT Background and Aims Hemophilia A and B are two of the most common bleeding disorders. Genetic risk factors are associated with the development of autoantibodies released in hemophilia patients against alternative factors and are the most important problems associated with the care of these patients. Objective In this study, we reviewed genetic
Zeylabi F, Aghaei M, Saki N.
europepmc   +2 more sources

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

Perinatal Gene Transfer to the Liver [PDF]

, 2011
The liver acts as a host to many functions hence raising the possibility that any one may be compromised by a single gene defect. Inherited or de novo mutations in these genes may result in relatively mild diseases or be so devastating that death within
Buckley, SM, Chan, JK, Howe, SJ, McKay, TR, Rahim, AA, Waddington, SN, Ward, NJ   +6 more
core   +1 more source

A review of evidence about behavioural and psychological aspects of chronic joint pain among people with haemophilia [PDF]

, 2014
Joint pain related to haemophilia affects large numbers of people and has a significant impact on their quality of life. This article reviews evidence about behavioural and psychological aspects of joint pain in haemophilia, and considers that evidence ...
Acharya, Barlow, Barry, Buhrman, Buzzard, Cour, Eckman, Elander, Elander, Elander, Elander, Elander, Ersek, Fischer, Fischer, Fishbain, Galen, Genderen, Gil, Hermans, Hilberg, Holstein, Humphries, Ingerslev, Jensen, Jensen, Karimi, Korff, Laforest, LeFort, Lorig, Lorig, Lorig, Lusher, Mackensen, McCracken, McCracken, McCracken, McCracken, Moore, Moore, Mulvany, Neufeld, Niscola, O'Mahony, Penica, Poon, Riley, Risdon, Rodriguez-Merchan, Royal, Santavirta, Santavirta, Schramm, Schulz, Sherry, Solomon, Solovieva, Spitzer, Stover, Swirsky-Sacchetti, Szende, Thorn, Turk, Turk, Varni, Varni, Varni, Varni, Wallny, Wilson, Witkop, Witkop, Wright, Zawilska, Zhou   +75 more
core   +2 more sources

Prophylaxis in Hemophilia

Clinical and Biomedical Research, 2023
Hemophilia is an inherited X-linked coagulopathy defined by a deficiency or abnormality in the clotting function of factor VIII (Hemophilia A) or factor IX (Hemophilia B).
Júlia Plentz Portich, Dayenne Catelli, Ebellins Tabares Calvache, Cristiane Seganfredo Weber, Daniel Sander Hoffmann, Guilherme Rasia Bosi, Leo Sekine, Lucia Mariano da Rocha Silla   +7 more
doaj  

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

Hemophilia: a biography on therapeutical approaches

Clinical and Biomedical Research, 2023
The history of hemophilia is ancient, with descriptions dated to the 2nd century AD. The first modern narratives appeared in 1800s, when total blood transfusion was the only available treatment and life expectancy was remarkably low.
Dayenne Catelli, Júlia Plentz Portich, Ebellins Tabares Calvache, Cristiane Seganfredo Weber, Daniel Sander Hoffmann, Guilherme Rasia Bosi, Leo Sekine, Lucia Mariano da Rocha Silla   +7 more
doaj  

Comorbidities, Health-Related Quality of Life, Health-care Utilization in Older Persons with Hemophilia—Hematology Utilization Group Study Part VII (HUGS VII)

Journal of Blood Medicine, 2022
Randall Curtis,1 Marilyn Manco-Johnson,2 Barbara A Konkle,3 Roshni Kulkarni,4 Joanne Wu,5 Judith R Baker,6 Megan Ullman,7 Duc Quang Tran Jr,8 Michael B Nichol5 1Factor VIII Computing, Berkeley, CA, USA; 2Hemophilia and Thrombosis Center, University of ...
Curtis R, Manco-Johnson M, Konkle BA, Kulkarni R, Wu J, Baker JR, Ullman M, Tran DQ Jr, Nichol MB   +8 more
doaj