PHILADELPHIA and GORDONVILLE, Pa. — Researchers from Children’s Hospital of Philadelphia (CHOP) and the Clinic for Special Children found that complement factor I (CFI) deficiency, an ultra-rare genetic disorder that can cause debilitating neuroinflammation, is more than 4500 times more likely to be found in individuals of Old Order Amish ancestry than the rest of the global population....
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Philadelphia, PA – Chimeric Antigen Receptors (CARs) have opened up an exciting new field of therapeutic advancements for rare and difficult-to-treat cancers, as they have the ability to deliver targeted therapies that can kill tumor cells. Peptide-centric CARs (PC-CARs) rely on specific peptide “barcodes,” which are derived from proteins within...
PHILADELPHIA, PA — Researchers at Children’s Hospital of Philadelphia (CHOP) and Children’s National Hospital in Washington D.C. are uncovering how rare inherited genetic variants contribute to the development of brain and spinal cord tumors in children. The findings, published in the journal Nature Communications, provide new insights into how a child’s genetic makeup...
Two new studies demonstrate the use of adeno-associated viral vectors for brain gene therapies PHILADELPHIA, PA — Adeno-associated viral (AAV) vectors are promising tools that can transport modified genetic material into the nuclei of cells in target tissues impacted by challenging diseases, offering the possibility of a one-time precision therapy...
PHILADELPHIA – Researchers at Children’s Hospital of Philadelphia (CHOP) have identified a key target that may be responsible for treatment failure in about 30% of patients with hemophilia A. The target, known as B cell activating factor (BAFF), appears to promote antibodies against and inhibitors of the missing blood clotting factor that...
Washington, DC – Trametinib, a mitogen-activated protein kinase (MEK) inhibitor, reduces mortality and morbidity in children with severe hypertrophic cardiomyopathy (HCM) caused by pathogenic variants in the RAS/MAPK pathway, according to a study published today in JACC: Basic to Translational Science. The study provides strong evidence for personalized treatment targeting...
Seoul, South Korea – To fill this knowledge gap, a group of researchers led by Professor Kangseok Lee, from the Department of Life Science, Chung-Ang University set out to explore the biogenesis and function of specific tRFs like tRHs. Their study published in Volume 15 of Nature Communications on 28...
City of Hope, one of the largest cancer research and treatment organizations in the United States and a leading research center for diabetes and other life-threatening illnesses, today announced that its Department of Stem Cell Biology and Regenerative Medicine received a $4.9 million grant to train the next generation of...
Duarte, CA – Scientists at City of Hope, one of the largest cancer research and treatment organizations in the United States, have discovered a new cellular mechanism that plays an important role in cancer cells’ ability to cause disease. The study was published in Nature Structural & Molecular Biology today....
LOS ANGELES, Calif. — City of Hope, one of the largest cancer research and treatment organizations in the United States, treated the oldest person to be cured of a blood cancer and then achieve remission for HIV after receiving a blood stem cell transplant from a donor with a rare...