Hemophilia | CHOP Research Institute


Published on
May 24, 2024
In this week’s news, our researchers have developed the first-of-its-kind bone marrow atlas and innovative, painless wound dressings. Read on to learn more.
Published on
Dec 28, 2023
These six stories received the highest number of engagements on CHOP Research social media channels in 2023.
Published on
Oct 10, 2023
CHOP's NoT Bleeding Program will conduct clinical trials to develop the next generation of therapeutics for hemophilia A, B, and other bleeding disorders.
Published on
Aug 29, 2023
The Clinical In Vivo Gene Therapy group supports CHOP’s continued leadership in the in vivo gene therapy space.
Published on
May 12, 2022
CHOP recognized gene therapy pioneer, Katherine High, MD, with Children’s Hospital of Philadelphia’s Gold Medal Award.

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

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.

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.

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.

samelsonjonesb [at] chop.edu
Published on
Apr 19, 2019
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.