Results 121 to 130 of about 22,597,991 (289)
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
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
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
Nature Inspired Delivery Vehicles for CRISPR‐Based Genome Editing
Small, EarlyView.The review highlights nature‐inspired nanocarriers for CRISPR delivery, emphasizing viral vectors, extracellular vesicles, liposomes, and lipid nanoparticles. It discusses their roles in improving specificity, minimizing immunogenicity, and overcoming barriers in genome editing. Recent advancements, challenges, and therapeutic applications are explored,
Elizabeth Maria Clarissa +4 more
wiley +1 more source
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
State of Iowa HIV and AIDS Surveillance Report, December 2004 [PDF]
, 2004 After taking a dip in 2003, HIV diagnoses were back up in 2004. There were 103 persons diagnosed in 2004, very close to our ten-year average of 100 cases per year. In 2003, there were 91 diagnoses.
core
Strategies for Hemophilia Treatment, a literature review of current evidence
مجله دانشگاه علوم پزشکی بیرجند, 2023 Hemophilia is an inherited bleeding disorder caused by malfunctioning or lacking blood coagulation factor VIII (hemophilia A) or IX (hemophilia B). Currently, the main treatments for these X-linked diseases are replacement therapy using periodic and ...
Fahimeh Ghasemi +3 more
doaj
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
Blood Advances, 2017 Emicizumab (ACE910), a recombinant humanized bispecific monoclonal antibody, provides factor VIII (FVIII) cofactor bridging function to restore hemostasis in people with hemophilia A.
M. Shima +9 more
semanticscholar +1 more source
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