Research News from CHOP

Web-Based Resource Provides Precise Classification of Dual Genome Variants of Primary Mitochondrial Disease
Quick-Mitome, developed by an international consortium of mitochondrial disease specialists, is available for non-commercial, non-clinical research.
CHOP Researchers Successfully Employ New Method for Treating Pediatric Patients with Common Heart Valve Disease
This new method, typically used in adult patients, may spare patients from having to undergo open heart surgery.
The PUSH Study: Patterns of Polysubstance Use and Its Association with Sexual Partnership Factors
Researchers from Children’s Hospital of Philadelphia (CHOP) and Johns Hopkins University School of Medicine studied how high levels of substance and polysubstance use are associated with sexual practices, and partner characteristics that can reinforce risk for HIV acquisition or transmission among Black and Latinx sexual youth as well as gender minority youth.

Children’s Hospital of Philadelphia Performs First in U.S. Gene Therapy Procedure to Treat Genetic Hearing Loss
Children’s Hospital of Philadelphia is proud to announce the initial results of an experimental gene therapy treatment of a patient with hereditary hearing loss in the United States. Findings to date indicate that the treatment was successful.
COVID-19 Vaccine Reduces Long COVID in Children
Vaccination associated with moderate protection in large, diverse cohort.
CHOP Researchers Develop Algorithm to Determine How Cellular “Neighborhoods” Function in Tissues
Data from this method could be used to discover how cancers are able to evade therapy and provide better insight into underlying tumor mechanisms.
Researchers from CHOP, Drexel Find Disparities in Rates of Certain Preterm Births
Medically indicated preterm births, like cesarean sections and induced births, can be a sign of other negative health issues affecting patients at neighborhood levels.
CHOP Researchers Find About a Quarter of Mitochondrial Disease Patients Suffer from Malnutrition
The study also found that increased fat and protein intake may improve muscle strength and quality of life in patients with mitochondrial disease.

FDA Approves Two Gene Therapies for Sickle Cell Disease
The Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel) and LYFGENIA™ (lovotibeglogene autotemcel), the first two gene therapies for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises (VOCs).
CHOP Researchers Discover Deep Structural Biology Connections that Help Improve CAR Therapy
Identifying “backbones” that link complexes can help maximize CAR therapy across different variants and tumor types.