An experimental gene therapy, studied in a preclinical model of Batten disease, was found to slow symptoms and extend lifespan

Researchers at Children’s Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania unlocked important new insights in how the immune system selects the right signals to alert T cells, a vital step in the prevention and treatment of serious illness. The findings were published in the journal Proceedings of the National Academy of Sciences (PNAS).

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Researchers at Children’s Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania developed a new screening technology, Aptamer-based T Lymphocyte Activity Screening and SEQuencing (ATLAS-seq), to better identify antigen-reactive T cells that are more likely to offer greater immune responses against cancer cells.

Researchers at Children’s Hospital of Philadelphia today announced results from the first ever international survey of trochleoplasty experts, which they hope will help guide current practice and future research regarding this procedure.

Researchers from the Children’s Hospital of Philadelphia (CHOP) in collaboration with the School of Veterinary Medicine at the University of Pennsylvania, Perelman School of Medicine at the University of Pennsylvania and Vanderbilt University Medical Center found that venous endothelial cells (VECs), a specific type of lung blood vessel cell, can help fix damaged blood vessels in the lungs following lung injury or disease. Their findings were recently published in Nature Cardiovascular Research.

Researchers at Children’s Hospital of Philadelphia (CHOP) and the University of Pennsylvania Perelman School of Medicine pioneered a first of its kind gene therapy model that offers a potential breakthrough in treating X-linked sideroblastic anemia (XLSA), a rare congenital anemia caused by mutations in the ALAS2 gene crucial for the synthesis of heme, a key compound in hemoglobin. This study marks the first time researchers studied gene therapy to treat this disease, which the authors underscore could have an impact on a broad spectrum of diseases. The research was published today and featured on the cover of the journal Blood.