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Researchers have successfully applied a potential gene therapy for hemophilia B based on the genome editing properties of the CRISPR/Cas9 system. The results of this study using mice were presented recently at the 58th Annual American Society of Hematology Meeting and Exposition in San Diego, Calif. Hemophilia B is a disease…
Recent evidence suggests that hemophilia B is clinically less severe than hemophilia A, highlighting the need to discuss further therapeutic options for each type of hemophilia. The study, “Haemophilia B is clinically less severe than haemophilia A: further evidence,” was published in Blood Transfusion. Hemophilia is an inherited, genetic…
Scientists have improved protein drugs using an approach based on “ancestral sequence reconstruction,” or ASR. The newly established strategy promises to enhance the ability to engineer more efficient and durable proteins, as in the case of human factor VIII for hemophilia. The study “Enhancing the pharmaceutical properties of protein drugs by…
A recent clinical trial indicates that replacement therapy with plasma-derived factor VIII is associated with lower incidence for development of inhibitors in patients with severe hemophilia A. The study,“A Randomized Trial of Factor VIII and Neutralizing Antibodies in Hemophilia A,” was published in The New England Journal of…
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