In this video, you'll learn how ex vivo gene therapy works and why it's bringing new hope to children with conditions like leukemia and sickle cell disease. CHOP was the first hospital in the world to use this groundbreaking therapy to treat a child with leukemia — and our researchers continue to pioneer new advances that are changing lives every day. At Children's Hospital of Philadelphia (CHOP), we're leading the way in gene therapy research and treatment so that more kids can have bright futures. Were you aware that there are two methods of VIVO Gene Therapy?

CRISPR-Cas9 technology is used to edit DNA to correct genetic abnormalities, but until now has required cells be removed for gene editing outside the body. In March 2020 researchers announced a test of the first in-the-body (in vivo) CRISPR-Cas9 treatment for Leber congenital amaurosis (LCA) type 10, a condition that causes severe, early-onset vision loss. This video explains the principles of DNA editing with #CRISPR-Cas9 and the rationale for its use and testing as a therapy for LCA10 in the BRILLIANCE clinical trial. Were you aware of IN VIVO Gene Therapies for the Eyes?

Solid Tumor Targeting: CAR-T cell therapies, which re-engineer a patient's immune cells, are being advanced to target solid tumors, which are generally more resistant to treatment than blood cancers.The Food and Drug Administration has approved the first treatment that genetically engineers patients' own blood cells into an army of assassins to seek and destroy childhood leukemia. Here's a look at how the powerful CAR-T cell therapy works. Were you aware of CAR T Cell Therapies?