Results for Coagulation Factor VIII

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Research Shows Multiyear Factor VIII Expression After Hemophilia A Gene Therapy

Published on
Nov 19, 2021
in Cornerstone Blog
A one-time treatment with an adeno-associated viral vector demonstrated safe, sustained factor VIII expression for patients with hemophilia A.
Dr. Lindsey George and colleagues report safe and sustained expression following hemophilia A gene therapy.

CHOP-led Study Shows Novel Gene Therapy for Hemophilia A Leads to Sustained Expression of Clotting Factor and Reduced Bleeding Events

Published on
Nov 17, 2021
in Press Releases
Results from multicenter study are first to show improved production of coagulation factor VIII over prolong period, reducing bleeding events.

In the News: 22q, Mentoring Award, Neonatal E. coli, Gene Therapy, Siegel Award

Published on
Nov 20, 2020
in Cornerstone Blog
In the News
This week in the news: New awards for our scientists and findings for antibiotic stewardship, gene therapy, and 22q11.2 deletion syndrome.

Pioneering Novel Therapies for Hemophilia

Published on
Sep 20, 2018
in Cornerstone Blog
hemophilia
Two exciting lines of research build on what we know about hemophilia to improve existing treatments.

Samelson-Jones Laboratory

Samelson-Jones Lab

Our lab is interested in the hemostasis system, especially coagulation factor VIII and IX, which are deficient in the bleeding disorders hemophilia A and B. By studying these blood proteins, the lab team aims to improve therapies for children with these diseases including gene therapy.

George Laboratory

George Labatory

Dr. George's clinical and research interests are in the development of novel therapeutics for hemophilia. The George Laboratory studies the molecular basis of coagulation with specific emphasis on the intrinsic tenase enzyme complex.

Novel Therapeutics for Bleeding Disorders

Novel Therapeutics for Bleeding Disorders

Combining our proven clinical expertise in hemophilia gene therapy and care of bleeding disorders with our research expertise to solidify Children’s Hospital of Philadelphia as the premier center for the therapeutic development and advanced clinical care of inherited bleeding disorders.

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