Gene Therapy | CHOP Research Institute
 

Gene Therapy

Published on
Jan 15, 2025
Researchers are creating a platform to help fast-track personalized gene-editing therapies for patients with urea cycle disorders.
Published on
Jan 14, 2025
In a preclinical model, the team reversed symptoms of X-linked sideroblastic anemia, a rare disorder.
Published on
Dec 30, 2024
We tallied up the most popular Cornerstone blog stories of 2024 based on data across our CHOP Research social media platforms.
Published on
Nov 22, 2024
Congratulations to physician scientist Dr. Yael Mosse, who received a Penn Medicine Award of Excellence for her contributions to pediatric oncology, and more.
Published on
Nov 20, 2024
Improving clinical care is at the heart of the Craniofacial Program, designated as a new Frontier Program at CHOP.
In this gene therapy study, we will treat children with deafness caused by an abnormal gene called otoferlin (OTOF).
Published on
Sep 27, 2024
Symposium highlights AAV gene therapy best practices for children, CHOP researchers develop novel nanopore platform, and more In the News
Published on
Sep 19, 2024
CHOP researchers developed a factor VIII variant that led to safe and long-lasting therapeutic results in preclinical models.

This study involves an investigational cellular therapy called Afamitresgene Autoleucel.

Published on
Jun 21, 2024
The Clinical Vector Core created a viral vector that changed the life of a child diagnosed with AADC deficiency.