In Vivo Gene Therapy | CHOP Research Institute
 

In Vivo Gene Therapy

Cornerstone Blog

How Can We Improve Gene Therapy to Treat Hemophilia A?

Published on
Sep 19, 2024
Red blood cells
CHOP researchers developed a factor VIII variant that led to safe and long-lasting therapeutic results in preclinical models.
Cornerstone Blog

Cell and Gene Therapy Collaborative Celebrates 5 Years of Ingenuity, Innovation

Published on
Jul 29, 2024
Cell and Gene Therapy Collaborative Celebrates 5 Years
The CGTC helps to accelerate clinical trials, training, and research, enabling CHOP to remain a global leader in the space.
Cornerstone Blog

Clinical Research Staff Shape the Success of Gene Therapy Trials

Published on
May 21, 2024
Clinical Research Staff Shape the Success of Gene Therapy Trials
As the administrative manager of the Clinical In Vivo Gene Therapy (CIGT) group, Sarah McCague manages a team of CRCs who are critical liaisons between families and the hospital.
Cornerstone Blog

A 30-Year Journey: How CHOP Researchers Designed the Gene Therapy Playbook

Published on
May 29, 2024
How CHOP Researchers Designed the Gene Therapy Playbook
Decades ago, Katherine A. High, MD, and colleagues began an unprecedented journey to develop a new kind of treatment for hemophilia B.

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.