In a preclinical study, researchers at Children’s Hospital of Philadelphia (CHOP) demonstrated a novel treatment strategy for patients with Fanconi anemia (FA), a rare genetic disease resulting in loss of blood-forming stem cells in the bone marrow. The researchers successfully used in situ mRNA delivered directly into the body by lipid nanoparticles (LNPs) to target bone marrow cells and transiently restore stem cell function in patients with FA.

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.

The vaccine is the first mRNA vaccine against C. difficile and would be the first vaccine in general to successfully ward off the bacterial infection.