Gene Therapy | CHOP Research Institute

Gene Therapy

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.
Published on
Jun 20, 2024
Using CRISPR/Cas9 gene editing in an early-stage clinical trial, researchers showed improved vision for a severe inherited form of blindness.
Published on
Jun 13, 2024
At CHOP’s Leukodystrophy Center, researchers are aiming to reduce the length of the prolonged diagnostic odyssey that many patients with MLD encounter.
Published on
May 21, 2024
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.
Published on
May 10, 2024
This week, honors for innovative CHOP investigators, early results of gene editing for inherited blindness, plus ASGCT and PAS meeting highlights.
Published on
May 29, 2024
Decades ago, Katherine A. High, MD, and colleagues began an unprecedented journey to develop a new kind of treatment for hemophilia B.
Published on
Apr 26, 2024
News highlights this week include the Biomedical Technician Training Program recognition event, new COVID-19 outcomes research, and more.
Published on
Mar 15, 2024
This week, CHOP announces two new Frontier Programs, and researchers define the origins of hematopoietic failure in Fanconi anemia.
Published on
Jan 23, 2024
This milestone represents an important step in treatment options for patients with hereditary hearing loss caused by genetic mutations.