Published on in CHOP News
In a proof-of-concept study, CHOP researchers used a vector to edit a single base mutation in Hurler Syndrome, halting progression of a disease that causes irreversible damage before birth.
Published on in CHOP News
CHOP researchers were interviewed as a part of a comprehensive article about cell and gene therapy in Philadelphia Magazine.
Published on in CHOP News
In a proof-of-concept study, CHOP and Penn researchers have identified ionizable lipid nanoparticles that could be used to deliver mRNA as part of fetal therapy.
Published on in CHOP News
CHOP researchers identified an in vivo role for activated protein C (APC), which could potentially be exploited for future hemophilia A treatments.
Published on in In Utero Insights
Prenatal correction of cleft lip and/or palate would offer benefits that are not attainable through conventional postnatal treatment. Learn about our work to correct it in the laboratory.
Published on in CHOP News
The National Academy of Medicine elects two global leaders in cellular and gene therapy from Children’s Hospital of Philadelphia.
Published on in Breakthrough Report
Two new treatments are giving children with spinal muscular atrophy hope. CHOP is on the forefront of delivering care that is changing children’s lives.
Published on in CHOP News
CHOP researchers use novel tool to mine clinical data and identify causative gene in severe childhood epilepsy.
Published on in CHOP News
Dr. George was recognized for the first gene therapy clinical trial to report a cure of hemophilia B patients.
Published on in Oncology Update, Cancer Connections
Trials of more game-changing cell therapies for pediatric cancer and other diseases are underway or on the horizon at Children's Hospital of Philadelphia.