Results 31 to 40 of about 13,243,158 (348)

Long‐term correction of hemophilia B using adenoviral delivery of CRISPR/Cas9 [PDF]

Journal of Controlled Release, 2019
C. Stephens, Elvin J Lauron, E. Kashentseva, Z. Lu, W. Yokoyama, D. Curiel   +5 more
semanticscholar   +3 more sources

CRISPR applications for Duchenne muscular dystrophy: From animal models to potential therapies

WIREs Mechanisms of Disease, Volume 15, Issue 1, January/February 2023., 2023
CRISPR‐Cas9 gene‐editing technology enables the rapid generation of animal models for Duchenne muscular dystrophy research and has potential to be developed as CRISPR therapy for the long lasting genetic correction of causal mutations. Abstract CRISPR gene‐editing technology creates precise and permanent modifications to DNA.
Yu C. J. Chey, Jayshen Arudkumar, Annemieke Aartsma‐Rus, Fatwa Adikusuma, Paul Q. Thomas   +4 more
wiley   +1 more source

Efficacy and safety in patients with haemophilia A switching to octocog alfa (BAY 81–8973): Final results of the global real‐world study, TAURUS

European Journal of Haematology, Volume 110, Issue 1, Page 77-87, January 2023., 2023
Abstract Objectives To report the final results of the 2‐year TAURUS study, assessing weekly prophylaxis dosing regimens of octocog alfa (Kovaltry®/BAY 81–8973) used in standard clinical practice in patients with moderate‐to‐severe haemophilia A. Methods TAURUS (NCT02830477) is a phase 4, multinational, prospective, non‐interventional, single‐arm study
Cristina Santoro, Beng Fuh, Phu Quoc Le, Philip Maes, Rubén Berrueco, Eva Maria Mingot‐Castellano, Sylvia von Mackensen, Claudia Tueckmantel, Jose Francisco Cabre‐Marquez, Michael Wang   +9 more
wiley   +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

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

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

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

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

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

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