Gene Therapy

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

Using CRISPR/Cas9 gene editing in an early-stage clinical trial, researchers showed improved vision for a severe inherited form of blindness.

At CHOP’s Leukodystrophy Center, researchers are aiming to reduce the length of the prolonged diagnostic odyssey that many patients with MLD encounter.

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.

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

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

News highlights this week include the Biomedical Technician Training Program recognition event, new COVID-19 outcomes research, and more.

This week, CHOP announces two new Frontier Programs, and researchers define the origins of hematopoietic failure in Fanconi anemia.

This milestone represents an important step in treatment options for patients with hereditary hearing loss caused by genetic mutations.