Gene Therapy

This edition highlights a study that suggests chronic illness may be on the rise for children in the U.S., a model of care that could improve postpartum health of mothers, and more.

A rapid genetic test developed at CHOP identified baby KJ’s rare mutation, setting the stage for a historic medical breakthrough.

The landmark study paves the way for researchers to customize the technology and treat other individual patients with genetic diseases caused by ultra-rare “N-of-1” variants.

In this Q&A, Dr. Kwiatkowski talks about her new clinical trial for a CHOP developed gene therapy for beta thalassemia.

STAT Madness selects CHOP-led research on hemophilia A gene therapy for the bracket competition, and two scientists are nominated as "Disruptors of the Year."

The Goldberg Lab is uncovering the mechanistic underpinnings of the disease, while studying a new form of genome editing as a potential fix.

Researchers are creating a platform to help fast-track personalized gene-editing therapies for patients with urea cycle disorders.

In a preclinical model, the team reversed symptoms of X-linked sideroblastic anemia, a rare disorder.

We tallied up the most popular Cornerstone blog stories of 2024 based on data across our CHOP Research social media platforms.

Congratulations to physician scientist Dr. Yael Mosse, who received a Penn Medicine Award of Excellence for her contributions to pediatric oncology, and more.