Pioneering new approaches to diagnosis, care, and treatment for patients with red blood cell disorders with novel gene therapies and stem-cell-based treatments that aim to reduce symptoms and prolong life.
When something important is missing, we often search for a replacement. After many years of looking, a team of researchers at The Children’s Hospital of Philadelphia and the University of Missouri have found a way to substitute for a missing gene linked to a relentless childhood neurodegenerative disease.
