Hemophilia | CHOP Research Institute

Cell and Gene Therapy Stories Lead Our Most Popular of 2023

Published on

Dec 28, 2023

in Cornerstone Blog

These six stories received the highest number of engagements on CHOP Research social media channels in 2023.

CHOP's NoT Bleeding Program Advancing Therapies for Hemophilia A and B

Published on

Oct 10, 2023

in Cornerstone Blog

Frontier Program Advancing Therapies for Hemophilia

CHOP's NoT Bleeding Program will conduct clinical trials to develop the next generation of therapeutics for hemophilia A, B, and other bleeding disorders.

CIGT Advances CHOP's In Vivo Gene Therapy Leadership

Published on

Aug 29, 2023

in Cornerstone Blog

CIGT Advances CHOP's In Vivo Gene Therapy Leadership

The Clinical In Vivo Gene Therapy group supports CHOP’s continued leadership in the in vivo gene therapy space.

Katherine High, MD, Awarded Esteemed CHOP Gold Medal

Published on

May 12, 2022

in Cornerstone Blog

Katherine High, MD, Awarded Esteemed CHOP Gold Medal

CHOP recognized gene therapy pioneer, Katherine High, MD, with Children’s Hospital of Philadelphia’s Gold Medal Award.

Gene Therapy Trial for Hemophilia A

This clinical trial study uses a modified adeno-associated virus (AAV) to carry the human factor VIII gene to the liver.

Sabatino Laboratory

The Sabatino Lab is focused on the inherited bleeding disorder, hemophilia. Our research interests include the study of variants of coagulation factor VIII to understand the biochemical properties of these proteins and to identify novel variants with enhanced function, and the development of gene-based therapeutic treatment approaches.

Samelson-Jones Laboratory

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.

Benjamin J. Samelson-Jones, MD, PhD

Dr. Samelson-Jones is a pediatric hematologist dedicated to improving the lives of children with bleeding and clotting disorders. His research focuses on gene therapy for hemophilia, the biochemical basis of coagulation, and the immune responses to hemophilia therapies.

E-mail:

samelsonjonesb [at] chop.edu

PAS 2019, NFL Helmet Safety, American Society of Gene and Cell Therapy, Bypass Prophylaxis Hemophilia, Lung Disease In Utero

Published on

Apr 19, 2019

in Cornerstone Blog

Whether they study helmets on the football field or hemophilia in a lab, our scientists at Children’s Hospital of Philadelphia Research Institute are always on the cutting-edge of their respective fields, as the latest roundup of research news shows.

Paris Margaritis, DPhil

Dr. Margaritis uses biochemical, molecular, and complex in vivo methodology within the field of coagulation to advance the understanding of molecular mechanisms involved in pro- and anti-coagulant reactions, and translate research for the treatment of coagulation defects.

E-mail:

margaritis [at] chop.edu