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MIS-C Patients Have Deeply Dysregulated Protein Profiles, According to New CHOP Research
CHOP researchers found that levels of proteins in the plasma of patients with MIS-C are significantly dysregulated and share features with two other inflammatory syndromes.
Children’s Hospital of Philadelphia Appoints New Chief of Hematology
Alexis Anne Thompson, MD, to fulfill new leadership role, bringing with her expertise in blood disorders and clinical practice.
CHOP-led Study Shows Novel Gene Therapy for Hemophilia A Leads to Sustained Expression of Clotting Factor and Reduced Bleeding Events
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.
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.
Kwaku Ohene-Frempong, MD Named a 2021 Honorific Award Recipient by the American Society of Hematology
Kwaku Ohene-Frempong, MD was named a 2021 Honorific Award Recipient by the American Society of Hematology.
CHOP Celebrates NASH Day 2021
The Non-Alcoholic Fatty Liver Disease (NAFLD) Clinic at Children’s Hospital of Philadelphia (CHOP) celebrates International NASH Day 2021.

CHOP Researchers Develop Proof-of-Concept Treatment that Elevates Both Adult and Fetal Hemoglobin
CHOP researchers have developed a proof-of-concept treatment for blood disorders like sickle cell disease that elevates both fetal and adult hemoglobin.
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-led Research Study Identifies Key Target in Treatment-Resistant Hemophilia A
CHOP researchers have identified BAFF as a key target that may be responsible for treatment failure in about 30% of patients with hemophilia A.
CHOP Researchers Find Ribosome Assembly Essential for Stem Cell Regeneration
The findings identify possible targets for future therapies for ribosomopathies, childhood disorders that lead to bone marrow failure (BMF).