Results from multicenter study are first to show improved production of coagulation factor VIII over prolonged period, leading to reduction or complete elimination of bleeding events.

CHOP researchers have developed a novel cancer therapy that targets proteins inside cancer cells that are essential for tumor growth and survival but have been historically impossible to reach.

A collaborative team of researchers, including Dr. Stephan Grupp, recently presented preliminary data showing that a CRISPR-based gene-editing therapy for inherited blood disorders is safe and effective.

CHOP researchers have developed a “dimmer switch” system based on alternative RNA splicing that can control levels of proteins expressed from gene therapy vectors.

The treatment modifies a domain in the CAR that targets CD19, making it look more human and therefore less likely to be rejected by patients.

On May 28, Dr. Stephan Grupp was featured on The Doctors to discuss the latest in CAR-T cell therapy and how it is changing the future of cancer care.

CHOP researchers were interviewed as a part of a comprehensive article about cell and gene therapy in Philadelphia Magazine.

CHOP researchers have developed a gene therapy vector for sickle cell disease and other blood disorder that is potentially superior to competitors.