Hemophilia | CHOP Research Institute

Cornerstone Blog

College of Physicians Induction, CGT Collaborative Awardees, Bone Marrow Atlas

Published on

May 24, 2024

College of Physicians Induction, CGT Collaborative Awardees

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.

Cornerstone Blog

Cell and Gene Therapy Stories Lead Our Most Popular of 2023

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Dec 28, 2023

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

Cornerstone Blog

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

Published on

Oct 10, 2023

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.

Cornerstone Blog

CIGT Advances CHOP's In Vivo Gene Therapy Leadership

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Aug 29, 2023

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.

Cornerstone Blog

Katherine High, MD, Awarded Esteemed CHOP Gold Medal

Published on

May 12, 2022

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

Cornerstone Blog

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

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.