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NIH Awards Penn Medicine and Children’s Hospital of Philadelphia $26 Million Grant to Develop Therapies for Rare Newborn Genetic Diseases
A Penn Medicine and CHOP team will seek to develop treatments for three rare, incurable genetic diseases with the help of a $26 million grant from the National Institutes of Health.
Uncovering Lysosomal Storage Diseases Beyond Newborn Screening
Jaden was a 4-year-old who presented to his PCP’s office with a persistent cough following an upper respiratory infection. Parents reported Jaden also had easy bruising, a symptom they had also noted after he had started walking.
CHOP Researchers Use Base Editing in Preclinical Model to Correct Lethal Lysosomal Storage Disease Before Birth
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.
Children’s Hospital of Philadelphia and Social Security Fund (CSS) in Panama Sign Letter of Understanding for Clinical Education
Children’s Hospital of Philadelphia (CHOP) and the Social Security Fund (CSS) in Panama have signed a letter of understanding to explore a collaborative training and educational program. The letter of understanding provides a high-level, comprehensive overview of potential collaborations that will enhance clinical insights and improve pediatric patient care.