Cell & Gene Therapy News

Positive Preliminary Data on CRISPR Treatment for Blood Diseases
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.
Children’s Hospital of Philadelphia Researchers Develop “Dimmer Switch” to Help Control Gene Therapy
CHOP researchers have developed a “dimmer switch” system based on alternative RNA splicing that can control levels of proteins expressed from gene therapy vectors.
CHOP Researchers Develop Humanized CAR T-Cell Therapy that Shows Potential for Patients with Relapsed B-ALL
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.
Dr. Stephan Grupp and Patient Emily Whitehead Featured on The Doctors
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 Gene Therapy Experts Profiled in Philadelphia Magazine
CHOP researchers were interviewed as a part of a comprehensive article about cell and gene therapy in Philadelphia Magazine.

CHOP Researchers Develop Potentially Safer, More Effective Gene Therapy Vector for Blood Disorders
CHOP researchers have developed a gene therapy vector for sickle cell disease and other blood disorder that is potentially superior to competitors.