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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.

From Mentors to Making it Your Own: Inspiration From Women in STEM

Published on
Mar 8, 2021
in Cornerstone Blog
Inspiration from Women in STEM at CHOP
Nominated by their Centers of Emphasis, women in STEM share what drives them, their words of wisdom, and why they do what they do.

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.

Camire Laboratory

Camire Lab

The Camire Lab is interested in understanding the components of the blood coagulation system, how they interface with activated cells, and how disturbances in their function lead to bleeding and thrombosis.

Sabatino Laboratory

Sabatino Lab

The research in the Sabatino Laboratory is focused on the inherited bleeding disorder, hemophilia.

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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  • (-) Raymond G. Perelman Center for Cellular and Molecular Therapeutics (7)
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  • Division of Hematology (7)
  • Clinical In Vivo Gene Therapy (3)
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  • Center for the Investigation of Factor FVIII Immunogenicity (1)
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  • George Laboratory (1)
  • Samelson-Jones Laboratory (1)

In this section

Organizations

  • Division of Hematology (7)
  • Clinical In Vivo Gene Therapy (3)
  • Department of Pediatrics (2)
  • Camire Laboratory (1)
  • Center for Childhood Cancer Research (1)
  • Center for Computational and Genomic Medicine (1)
  • Center for the Investigation of Factor FVIII Immunogenicity (1)
  • Clinical Futures (1)
  • Division of Human Genetics (1)
  • Division of Neonatology (1)
  • Division of Protective Immunity (1)
  • Division of Pulmonary and Sleep Medicine (1)
  • George Laboratory (1)
  • Samelson-Jones Laboratory (1)
  • (-) Raymond G. Perelman Center for Cellular and Molecular Therapeutics (7)
  • (-) Center for Injury Research and Prevention (1)
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