Clinical In Vivo Gene Therapy
The Clinical In Vivo Gene Therapy (CIGT) group is dedicated to advancing clinical in vivo gene therapy at Children’s Hospital of Philadelphia (CHOP). Through industry partnerships and investigator-initiated trials, CIGT seeks to further the development of transformative human therapies and to build upon CHOP’s legacy as a leader in gene therapy.
CHOP has been at the forefront of major in vivo gene therapy milestones over the past two decades. To date, we have treated more than 120 patients from 15 countries with in vivo gene therapies. Led by experts with more than 10 years of gene therapy clinical trial experience, the CIGT team aims to offer more breakthrough therapies to our patients sooner, by streamlining the startup of complex gene therapy trials and ensuring licensed gene therapies are available in our clinics.
Conditions We Treat
- Choroideremia
- Danon disease
- Duchenne muscular dystrophy (DMD)
- GM1 gangliosidosis
- Hemophilia A
- Hemophilia B
- Inherited retinal dystrophy
- Krabbe disease
- Leber congenital amaurosis
- Spinal muscular atrophy (SMA)
- Type 2 spinal muscular dystrophy
Related Programs and Services
Meet Our Team
Led by experts with more than 10 years of gene therapy clinical trial experience, the CIGT team aims to offer more breakthrough therapies to our patients sooner, by streamlining the startup of complex gene therapy trials and ensuring licensed gene therapies are available in our clinics.