Results 11 to 20 of about 83,381 (315)

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

Report on diagnosis and treatment of hemophilia in China 2023 [PDF]

Zhenduanxue lilun yu shijian, 2023
In recent years, China attaches great importance to the prevention and treatment of rare diseases. As one of the representative diseases of rare diseases, management of Hemophilia has made great progress in China.
XUE Feng, DAI Jing, CHEN Lixia, LIU Wei, ZHANG Houqiang, WU Runhui, SUN Jing, ZHANG Xinsheng, WU Jingsheng, ZHAO Yongqiang, WANG Xuefeng, YANG Renchi
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

CHARACTERIZATION AND MANAGEMENT OF PATIENTS WITH HEREDITARY FACTOR X DEFICIENCY: A RETROSPECTIVE SINGLE CENTER EXPERIENCE

Hematology, Transfusion and Cell Therapy, 2023
Objective: Factor X deficiency (FXd) is a rare coagulation disorder that can be either hereditary or acquired. Case report: We characterized patients with FXd and evaluated their bleeding patterns and treatment strategies. Methodology: This retrospective
Nigar Abdullayeva, Fahri Sahin, Zuhal Demirci, Bahar Sevgili   +3 more
doaj   +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

Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA. [PDF]

, 2001
Long-term expression of coagulation factor IX (FIX) has been observed in murine and canine models following administration of recombinant adeno-associated viral (rAAV) vectors into either the portal vein or muscle. These studies were designed to evaluate
Davidoff, A, Hanawa, H, Nathwani, AC, Nienhuis, AW, Vanin, EF, Zhou, JF   +5 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 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

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

Clinical, instrumental, serological and histological findings suggest that hemophilia B may be less severe than hemophilia A

Haematologica, 2016
Recent evidence suggests that patients with severe hemophilia B may have a less severe disease compared to severe hemophilia A. To investigate clinical, radiological, laboratory and histological differences in the arthropathy of severe hemophilia A and ...
Daniela Melchiorre, Silvia Linari, Mirko Manetti, Eloisa Romano, Francesco Sofi, Marco Matucci-Cerinic, Christian Carulli, Massimo Innocenti, Lidia Ibba-Manneschi, Giancarlo Castaman   +9 more
doaj   +1 more source