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Medical News Today: Mutant protein brings gene therapy for hemophilia a step closer

Health : Medical News Today

A mutant protein called FIX-Padua brings gene therapy for hemophilia - an inherited blood disorder - a step closer after it was found to ease symptoms in dogs with the disease.

One-Dose Gene Therapy Produces Clotting Factor, Safely Stops Bleeding in Hemophilia B Patients

Health : Newswise Medical News

A team of gene therapy researchers has reported positive results in a phase 1/2 clinical trial for the inherited bleeding disorder hemophilia B. A single intravenous infusion of a novel bioengineered gene therapy treatment enabled a...

Gene Therapy Shows Promise as Hemophilia Treatment in Animal Studies

Health : Newswise Medical News

For the first time, researchers have combined gene therapy and stem cell transplantation to successfully reverse the severe, crippling bleeding disorder hemophilia A in large animals, opening the door to the development of new thera...

Gene therapy study produces promising results for patients with hemophilia B

Health : Medical News Today

A fraction of patients with a common form of the bleeding disorder hemophilia develop an allergic reaction to the blood-clotting treatment they need to keep them alive. But using gene therapy, University of Florida researchers were ...

Genome Editing, a Next Step in Genetic Therapy, Corrects Hemophilia in Animals

Health : Newswise Medical News

Using an innovative, precise gene therapy tool called genome editing, scientists treated the blood clotting disorder hemophilia in mice. It's the first time in vivo genome editing has achieved clinically meaningful results.


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