Results 121 to 130 of about 22,643,381 (355)
PRODUCTION OF HIGH LEVELS OF TRANSGENC FACTOR IX WITHOUT GENE RESCUE, AND ITS THERAPEUTIC USES [PDF]
, 2011 A non-human transgenic mammalian animal, as described above, contains an exogenous double stranded DNA sequence stably integrated into the genome of the animal, which comprises cis-acting regulatory units operably linked to a DNA sequence encoding human ...
Cooper, Julian D. +2 more
core +1 more source
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
Curing hemophilia A by NHEJ-mediated ectopic F8 insertion in the mouse
Genome Biology, 2019 Hemophilia A, a bleeding disorder resulting from F8 mutations, can only be cured by gene therapy. A promising strategy is CRISPR-Cas9-mediated precise insertion of F8 in hepatocytes at highly expressed gene loci, such as albumin (Alb). Unfortunately, the
Jian-Ping Zhang +21 more
semanticscholar +1 more source
Small, EarlyView.Inspired by the Ampullae of Lorenzini in elasmobranch fish, a biomimetic electroreceptor platform is developed using an electret layer‐coated single‐walled carbon nanotube thin‐film transistor (eSWCNT‐TFT). This platform enables non‐contact, label‐free, and real‐time monitoring of DNA encapsulation efficiency in liposome droplets by detecting net ...
Younsu Jung +11 more
wiley +1 more source
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 +11 more
core +1 more source
Review Dental Treatment of Patients with Congenital Bleeding Disorders
Česká Stomatologie a Praktické Zubní Lékařství, 2014 Objectives: The commonest congenital bleeding disorders in childhood are hemophilia, von Willebrand disease and deficiency of factor XI. Disease is characterized by a deficient of coagulability. It is a gonosomal recessive condition.
J. Papež, K. Chleborád, T. Dostálová
doaj +1 more source
Gene therapy in hemophilia A: a cost-effectiveness analysis.
Blood Advances, 2018 Gene therapy provides a potential phenotypic cure for hemophilia, yet the cost of this novel treatment is high, tempering enthusiasm and raising questions regarding cost vs benefit.
Nicoletta Machin +2 more
semanticscholar +1 more source
Small, EarlyView.This review presents a detailed overview of clinically approved nanoparticle therapeutics, classifying them by type and discussing their unique advantages in drug delivery. It highlights regulatory challenges across global markets and emphasizes the need for adaptive approval pathways.
Nimeet Desai +5 more
wiley +1 more source
Journal of Blood Medicine, 2021 Amy D Shapiro,1 Alejandro Fernandez,2 Jerome Teitel,3 Jaco Botha,2 Kate Khair4 1Indiana Hemophilia & Thrombosis Center, Indianapolis, IN, USA; 2Takeda Pharmaceutical International AG, Zürich, Switzerland; 3St.
Shapiro AD +4 more
doaj
Acquired hemophilia A: a review of recent data and new therapeutic options
Hematology, 2017 Objectives: Acquired hemophilia A (AHA) is a rare, but potentially life-threatening, bleeding disorder caused by an autoantibody against factor VIII that interferes with its coagulant function.
M. Franchini +6 more
semanticscholar +1 more source