First Child to Receive Revolutionary CAR T Therapy Celebrates 10 Years Cancer Free
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Emily Whitehead, now 17, received life-saving treatment at CHOP, transforming the field of cancer immunotherapy.
Cell and Gene Therapy Collaborative News and Updates
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CHOP Brings Together Experts to Discuss Precision Medicine in Pediatric Cancer Care
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More than 250 experts gathered in Philadelphia and virtually to discuss the latest scientific advancements and breakthroughs in pediatric precision medicine oncology.
CHOP/Penn Study Shows CAR-T Cells Remain Active a Decade After Infusion
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A new study in Nature has documented the longest persistence of CAR T cell therapy to date.
Aberrant Splicing of CD22 in Acute Lymphoblastic Leukemia Underlies Resistance to Immunotherapy
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Aberrant splicing leads to downregulation of CD22 in pediatric B-ALL, rendering malignant cells resistant to the effects of CD22-directed immunotherapies.
CHOP-led Study Shows Novel Gene Therapy for Hemophilia A Leads to Sustained Expression of Clotting Factor and Reduced Bleeding Events
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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 Develop a New Class of CAR-T Cells that Target Previously Untargetable Cancer Drivers
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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.
Positive Preliminary Data on CRISPR Treatment for Blood Diseases
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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
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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 Use Base Editing in Preclinical Model to Correct Lethal Lysosomal Storage Disease Before Birth
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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.
CHOP Gene Therapy Experts Profiled in Philadelphia Magazine
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CHOP researchers were interviewed as a part of a comprehensive article about cell and gene therapy in Philadelphia Magazine.