Results 21 to 30 of about 13,000,271 (286)

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

The Cost-effectiveness of Gene Therapy for Severe Hemophilia B: Microsimulation Study from the United States Perspective.

Blood, 2021
Adeno-associated virus (AAV)-mediated gene therapy is a novel treatment promising to reduce morbidity associated with hemophilia. While multiple clinical trials continue to evaluate efficacy and safety, limited cost-effectiveness data have been published.
N. Bolous, Yichen Chen, Huiqi Wang, A. Davidoff, M. Devidas, Timothy W. Jacobs, M. Meagher, A. Nathwani, E. Neufeld, Bryan A. Piras, C. Rodríguez-Galindo, U. Reiss, N. Bhakta   +12 more
semanticscholar   +1 more source

Health care resource utilization and cost burden of hemophilia B in the United States.

Blood Advances, 2021
Hemophilia B is a rare congenital blood disorder characterized by factor IX deficiency. Clinical profiles of hemophilia B range from mild to severe forms of the disease.
T. Buckner, I. Bocharova, K. Hagan, A. Bensimon, Hongbo Yang, E. Wu, E. Sawyer, Nanxin Li   +7 more
semanticscholar   +1 more source

MOLECULAR MECHANISMS OF INHIBITOR DEVELOPMENT IN HEMOPHILIA

Mediterranean Journal of Hematology and Infectious Diseases, 2020
The development of neutralizing antibodies in hemophilia is a serious complication of factor replacement therapy. These antibodies, also known as “inhibitors”, significantly increase morbidity within the hemophilia population and lower the quality of ...
Davide Matino, Paul Tieu, Dr., Antony Chan   +2 more
doaj   +1 more source

Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.

New England Journal of Medicine, 2011
BACKGROUND Hemophilia B, an X-linked disorder, is ideally suited for gene therapy. We investigated the use of a new gene therapy in patients with the disorder.
A. Nathwani, E. Tuddenham, S. Rangarajan, Cecilia Rosales, J. McIntosh, D. Linch, P. Chowdary, A. Riddell, Arnulfo Pie, C. Harrington, J. O’Beirne, Keith A. Smith, J. Pasi, B. Glader, P. Rustagi, C. Ng, M. Kay, Junfang Zhou, Yunyu Spence, C. Morton, J. Allay, J. Coleman, S. Sleep, J. Cunningham, D. Srivastava, E. Basner-Tschakarjan, F. Mingozzi, K. High, J. Gray, U. Reiss, A. Nienhuis, A. Davidoff   +31 more
semanticscholar   +1 more source

Identification and Genetic Analysis of a Factor IX Gene Intron 3 Mutation in a Hemophilia B Pedigree in China

Turkish Journal of Hematology, 2014
OBJECTIVE: Hemophilia B is caused by coagulation defects in the factor IX gene located in Xq27.1 on the X chromosome. A wide range of mutations, showing extensive molecular heterogeneity, have been described in hemophilia B patients.
Dong Hua Cao, Xiao Li Liu, Kai Mu, Xiang Wei Ma, Jing Li Sun, Xiao Zhong Bai, Chang Kun Lin, Chun Lian Jin   +7 more
doaj   +1 more source

BAX 335 hemophilia B gene therapy clinical trial results - potential impact of CpG sequences on gene expression.

Blood, 2020
Gene therapy has the potential to maintain therapeutic blood clotting factor IX (FIX) levels in patients with hemophilia B by delivering a functional human F9 gene into liver cells.
B. Konkle, C. Walsh, Miguel A. Escobar, Neil C Josephson, G. Young, Annette von Drygalski, S. McPhee, R. Samulski, I. Bilic, Maurus de la Rosa, B. Reipert, H. Rottensteiner, F. Scheiflinger, J. Chapin, Bruce M. Ewenstein, P. Monahan   +15 more
semanticscholar   +1 more source

A Case of Hemophilia B with Abdominal and Pelvic Pseudotumor

罕见病研究, 2022
Hemophilia B is a genetic disorder caused by coagulation factor Ⅸ(FⅨ) deficiency, mainly manifesting as joint, muscle and deep tissue bleeding. Hemophilia pseudotumor is a mass formed by soft tissue liquefaction and necrosis caused by repeated bleeding ...
ZHANG Jing, JU Mankai, ZHANG Kemin, CHEN Lingling, WANG Yuhua, ZHANG Lei, YANG Renchi, XUE Feng   +7 more
doaj   +1 more source

Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

New England Journal of Medicine, 2014
BACKGROUND In patients with severe hemophilia B, gene therapy that is mediated by a novel self-complementary adeno-associated virus serotype 8 (AAV8) vector has been shown to raise factor IX levels for periods of up to 16 months.
A. Nathwani, U. Reiss, E. Tuddenham, Cecilia Rosales, P. Chowdary, J. McIntosh, M. della Peruta, Elsa Lhériteau, Nishali Patel, D. Raj, A. Riddell, J. Pie, S. Rangarajan, D. Bevan, M. Recht, Yu-min P Shen, K. Halka, E. Basner-Tschakarjan, F. Mingozzi, K. High, J. Allay, M. Kay, C. Ng, Junfang Zhou, M. Cancio, C. Morton, J. Gray, D. Srivastava, A. Nienhuis, A. Davidoff   +29 more
semanticscholar   +1 more source

Inhibitor incidence in an unselected cohort of previously untreated patients with severe hemophilia B: a PedNet study

Haematologica, 2020
The incidence of factor IX (FIX) inhibitors in severe hemophilia B (SHB) is not well defined. Frequencies of 3-5% have been reported but most studies to date have been small, including patients with different severities, and without prospective follow up
C. Male, N. Andersson, A. Rafowicz, R. Liesner, K. Kurnik, K. Fischer, H. Platokouki, E. Santagostino, H. Chambost, B. Nolan, C. Königs, G. Kenet, R. Ljung, M. Van den berg   +13 more
semanticscholar   +1 more source