Clinical Vector Core News & Announcements

CHOP researchers developed a factor VIII variant that led to safe and long-lasting therapeutic results in preclinical models.

The Clinical Vector Core created a viral vector that changed the life of a child diagnosed with AADC deficiency.

Decades ago, Katherine A. High, MD, and colleagues began an unprecedented journey to develop a new kind of treatment for hemophilia B.

This week, honors for innovative CHOP investigators, early results of gene editing for inherited blindness, plus ASGCT and PAS meeting highlights.

The Clinical In Vivo Gene Therapy group supports CHOP’s continued leadership in the in vivo gene therapy space.

This week’s research news roundup includes accolades for our scientists and the spaces they work in.

In this Q&A, Stephan Grupp, MD, PhD, discusses his experience delivering CAR T-cell therapy to the first pediatric patient, Emily Whitehead.

Dr. Lindsey George and colleagues report safe and sustained expression following hemophilia A gene therapy.

The Clinical Vector Core, an award-winning, state-of-the-art, multi-product GMP manufacturing facility, supports the growing field of gene therapy.

Save the Date for Virtual Cores Day 2021

This week’s news round-up highlights the CCMT’s state-of-the-art Clinical Manufacturing Facility

If there’s an injury on the mountain, these EMTs-on-skis come to the rescue. Han van der Loo, PhD, volunteers with the National Ski Patrol.

Before she was 7, Hannah had never seen a star. Then she had a breakthrough treatment at Children's Hospital of Philadelphia. Our donors help make breakthroughs like this happen.

In this week's roundup, CHOP researchers help parents avoid unnecessary ER visits, and share real-world implications of injury prevention work.

Meet two of the scientists behind the game-changing gene therapies created by the Research Institute's Clinical Vector Core.